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Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.

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Title: Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.
Authors: von Drygalski A; Division of Hematology/Oncology, Department of Medicine, University of California San Diego, San Diego, CA., Gomez E; Center for Inherited Blood Disorders, Orange, CA., Giermasz A; Division of Hematology/Oncology, Department of Medicine, Hemophilia Treatment Center, University of California Davis, Sacramento, CA., Castaman G; Center for Bleeding Disorders, Department of Oncology, Careggi University Hospital, Florence, Italy., Key NS; Division of Hematology and Blood Research Center, Department of Medicine, University of North Carolina, Chapel Hill, NC., Lattimore SU; Department of Pediatrics, The Hemostasis and Thrombosis Center, Oregon Health & Science University, Portland, OR., Leebeek FWG; Department of Hematology, Erasmus Medical Centre, University Medical Center, Rotterdam, The Netherlands., Miesbach WA; Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany., Recht M; National Bleeding Disorders Foundation, New York, NY.; Division of Pediatric Hematology-Oncology, Center for Bleeding and Clotting Disorders, Yale School of Medicine, New Haven, CT., Monahan PE; Hematology Clinical Development, CSL Behring, King of Prussia, PA., Le Quellec S; Hematology Clinical Development, CSL Behring, King of Prussia, PA., Pipe SW; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI.
Source: Blood advances [Blood Adv] 2025 Jul 22; Vol. 9 (14), pp. 3543-3552.
Publication Type: Journal Article; Clinical Trial, Phase II; Multicenter Study
Journal Info: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.
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  Data: <searchLink fieldCode="AU" term="%22von+Drygalski+A%22">von Drygalski A</searchLink>; Division of Hematology/Oncology, Department of Medicine, University of California San Diego, San Diego, CA.<br /><searchLink fieldCode="AU" term="%22Gomez+E%22">Gomez E</searchLink>; Center for Inherited Blood Disorders, Orange, CA.<br /><searchLink fieldCode="AU" term="%22Giermasz+A%22">Giermasz A</searchLink>; Division of Hematology/Oncology, Department of Medicine, Hemophilia Treatment Center, University of California Davis, Sacramento, CA.<br /><searchLink fieldCode="AU" term="%22Castaman+G%22">Castaman G</searchLink>; Center for Bleeding Disorders, Department of Oncology, Careggi University Hospital, Florence, Italy.<br /><searchLink fieldCode="AU" term="%22Key+NS%22">Key NS</searchLink>; Division of Hematology and Blood Research Center, Department of Medicine, University of North Carolina, Chapel Hill, NC.<br /><searchLink fieldCode="AU" term="%22Lattimore+SU%22">Lattimore SU</searchLink>; Department of Pediatrics, The Hemostasis and Thrombosis Center, Oregon Health & Science University, Portland, OR.<br /><searchLink fieldCode="AU" term="%22Leebeek+FWG%22">Leebeek FWG</searchLink>; Department of Hematology, Erasmus Medical Centre, University Medical Center, Rotterdam, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Miesbach+WA%22">Miesbach WA</searchLink>; Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany.<br /><searchLink fieldCode="AU" term="%22Recht+M%22">Recht M</searchLink>; National Bleeding Disorders Foundation, New York, NY.; Division of Pediatric Hematology-Oncology, Center for Bleeding and Clotting Disorders, Yale School of Medicine, New Haven, CT.<br /><searchLink fieldCode="AU" term="%22Monahan+PE%22">Monahan PE</searchLink>; Hematology Clinical Development, CSL Behring, King of Prussia, PA.<br /><searchLink fieldCode="AU" term="%22Le+Quellec+S%22">Le Quellec S</searchLink>; Hematology Clinical Development, CSL Behring, King of Prussia, PA.<br /><searchLink fieldCode="AU" term="%22Pipe+SW%22">Pipe SW</searchLink>; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI.
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  Data: <searchLink fieldCode="JN" term="%22101698425%22">Blood advances</searchLink> [Blood Adv] 2025 Jul 22; Vol. 9 (14), pp. 3543-3552.
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  Data: Journal Article; Clinical Trial, Phase II; Multicenter Study
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22American+Society+of+Hematology%22">American Society of Hematology </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101698425 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>2473-9537 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2224739529%22">24739529 </searchLink><i>NLM ISO Abbreviation: </i>Blood Adv <i>Subsets: </i>MEDLINE
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              Text: 2025 Jul 22
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