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

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Bibliographic Details
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
Description
ISSN:2473-9537
DOI:10.1182/bloodadvances.2024015291