Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
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| Title: | Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. |
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| Authors: | Pipe SW; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Leebeek FWG; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Recht M; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Key NS; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Castaman G; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Miesbach W; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Lattimore S; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Peerlinck K; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Van der Valk P; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Coppens M; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Kampmann P; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Meijer K; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., O'Connell N; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Pasi KJ; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Hart DP; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Kazmi R; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Astermark J; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Hermans CRJR; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Klamroth R; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Lemons R; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Visweshwar N; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., von Drygalski A; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Young G; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Crary SE; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Escobar M; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Gomez E; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Kruse-Jarres R; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Quon DV; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Symington E; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Wang M; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Wheeler AP; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Gut R; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Liu YP; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Dolmetsch RE; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Cooper DL; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Li Y; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Goldstein B; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.)., Monahan PE; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.). |
| Source: | The New England journal of medicine [N Engl J Med] 2023 Feb 23; Vol. 388 (8), pp. 706-718. |
| Publication Type: | Clinical Trial, Phase III; Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 36812434 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
| IllustrationInfo | |
| Items | – Name: Title Label: Title Group: Ti Data: Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Pipe+SW%22">Pipe SW</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Leebeek+FWG%22">Leebeek FWG</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Recht+M%22">Recht M</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Key+NS%22">Key NS</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Castaman+G%22">Castaman G</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Miesbach+W%22">Miesbach W</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Lattimore+S%22">Lattimore S</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Peerlinck+K%22">Peerlinck K</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Van+der+Valk+P%22">Van der Valk P</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Coppens+M%22">Coppens M</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Kampmann+P%22">Kampmann P</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Meijer+K%22">Meijer K</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22O'Connell+N%22">O'Connell N</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Pasi+KJ%22">Pasi KJ</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Hart+DP%22">Hart DP</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Kazmi+R%22">Kazmi R</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Astermark+J%22">Astermark J</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Hermans+CRJR%22">Hermans CRJR</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Klamroth+R%22">Klamroth R</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Lemons+R%22">Lemons R</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Visweshwar+N%22">Visweshwar N</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22von+Drygalski+A%22">von Drygalski A</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Young+G%22">Young G</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Crary+SE%22">Crary SE</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Escobar+M%22">Escobar M</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Gomez+E%22">Gomez E</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Kruse-Jarres+R%22">Kruse-Jarres R</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Quon+DV%22">Quon DV</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Symington+E%22">Symington E</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Wang+M%22">Wang M</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Wheeler+AP%22">Wheeler AP</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Gut+R%22">Gut R</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Liu+YP%22">Liu YP</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Dolmetsch+RE%22">Dolmetsch RE</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Cooper+DL%22">Cooper DL</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Li+Y%22">Li Y</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Goldstein+B%22">Goldstein B</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).<br /><searchLink fieldCode="AU" term="%22Monahan+PE%22">Monahan PE</searchLink>; From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.). – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2023 Feb 23; Vol. 388 (8), pp. 706-718. – Name: TypePub Label: Publication Type Group: TypPub Data: Clinical Trial, Phase III; Journal Article; Research Support, Non-U.S. Gov't – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Massachusetts+Medical+Society%22">Massachusetts Medical Society </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>0255562 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1533-4406 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200284793%22">00284793 </searchLink><i>NLM ISO Abbreviation: </i>N Engl J Med <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=36812434 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1056/NEJMoa2211644 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 706 Titles: – TitleFull: Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Pipe SW – PersonEntity: Name: NameFull: Leebeek FWG – PersonEntity: Name: NameFull: Recht M – PersonEntity: Name: NameFull: Key NS – PersonEntity: Name: NameFull: Castaman G – PersonEntity: Name: NameFull: Miesbach W – PersonEntity: Name: NameFull: Lattimore S – PersonEntity: Name: NameFull: Peerlinck K – PersonEntity: Name: NameFull: Van der Valk P – PersonEntity: Name: NameFull: Coppens M – PersonEntity: Name: NameFull: Kampmann P – PersonEntity: Name: NameFull: Meijer K – PersonEntity: Name: NameFull: O'Connell N – PersonEntity: Name: NameFull: Pasi KJ – PersonEntity: Name: NameFull: Hart DP – PersonEntity: Name: NameFull: Kazmi R – PersonEntity: Name: NameFull: Astermark J – PersonEntity: Name: NameFull: Hermans CRJR – PersonEntity: Name: NameFull: Klamroth R – PersonEntity: Name: NameFull: Lemons R – PersonEntity: Name: NameFull: Visweshwar N – PersonEntity: Name: NameFull: von Drygalski A – PersonEntity: Name: NameFull: Young G – PersonEntity: Name: NameFull: Crary SE – PersonEntity: Name: NameFull: Escobar M – PersonEntity: Name: NameFull: Gomez E – PersonEntity: Name: NameFull: Kruse-Jarres R – PersonEntity: Name: NameFull: Quon DV – PersonEntity: Name: NameFull: Symington E – PersonEntity: Name: NameFull: Wang M – PersonEntity: Name: NameFull: Wheeler AP – PersonEntity: Name: NameFull: Gut R – PersonEntity: Name: NameFull: Liu YP – PersonEntity: Name: NameFull: Dolmetsch RE – PersonEntity: Name: NameFull: Cooper DL – PersonEntity: Name: NameFull: Li Y – PersonEntity: Name: NameFull: Goldstein B – PersonEntity: Name: NameFull: Monahan PE IsPartOfRelationships: – BibEntity: Dates: – D: 23 M: 02 Text: 2023 Feb 23 Type: published Y: 2023 Identifiers: – Type: issn-electronic Value: 1533-4406 Numbering: – Type: volume Value: 388 – Type: issue Value: 8 Titles: – TitleFull: The New England journal of medicine Type: main |
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