Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.

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Title: Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.
Authors: Leavitt AD; Adult Hemophilia Treatment Center, Department of Medicine, University of California San Francisco, San Francisco, California, USA., Mahlangu J; Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa., Raheja P; The Royal London Hospital Haemophilia Centre, Barts Health NHS Trust, London, UK., Symington E; Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Quon DV; Orthopaedic Hemophilia Treatment Center, Los Angeles, California, USA., Giermasz A; Hemophilia Treatment Center, University of California Davis, Sacramento, California, USA., Kenet G; The National Hemophilia Center and Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Hashomer, Tel Aviv University, Tel Aviv, Israel., Lowe G; West Midlands Adult Haemophilia Comprehensive Care Centre, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK., Key NS; UNC Blood Research Center, University of North Carolina, Chapel Hill, North Carolina, USA., Millar CM; Centre for Haematology, Imperial College London, London, UK.; Imperial College Healthcare NHS Trust, London, UK., Pipe SW; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA., Chou SC; Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan., Klamroth R; Vascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin, Germany.; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany., Mason J; Queensland Haemophilia Centre, Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia.; University of Queensland, Brisbane, Queensland, Australia., Chambost H; AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France., Peyvandi F; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy., Majerus E; Department of Medicine, Washington University in St Louis, St Louis, Missouri, USA., Pepperell D; Department of Haematology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia., Tan CW; Royal Adelaide Hospital, Adelaide, South Australia, Australia., Yu H; BioMarin Pharmaceutical Inc, Novato, California, USA., Chavele KM; BioMarin UK Ltd, London, UK., Ozelo MC; Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Sao Paulo, Brazil.
Source: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2026 Mar 27; Vol. 10 (3), pp. 103416. Date of Electronic Publication: 2026 Mar 27 (Print Publication: 2026).
Publication Type: Journal Article; Clinical Trial, Phase III; Multicenter Study
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 101703775 Publication Model: eCollection Cited Medium: Internet ISSN: 2475-0379 (Electronic) Linking ISSN: 24750379 NLM ISO Abbreviation: Res Pract Thromb Haemost Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2475-0379
DOI:10.1016/j.rpth.2026.103416