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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
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  Data: 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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  Data: <searchLink fieldCode="AU" term="%22Leavitt+AD%22">Leavitt AD</searchLink>; Adult Hemophilia Treatment Center, Department of Medicine, University of California San Francisco, San Francisco, California, USA.<br /><searchLink fieldCode="AU" term="%22Mahlangu+J%22">Mahlangu J</searchLink>; Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.<br /><searchLink fieldCode="AU" term="%22Raheja+P%22">Raheja P</searchLink>; The Royal London Hospital Haemophilia Centre, Barts Health NHS Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Symington+E%22">Symington E</searchLink>; Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Quon+DV%22">Quon DV</searchLink>; Orthopaedic Hemophilia Treatment Center, Los Angeles, California, USA.<br /><searchLink fieldCode="AU" term="%22Giermasz+A%22">Giermasz A</searchLink>; Hemophilia Treatment Center, University of California Davis, Sacramento, California, USA.<br /><searchLink fieldCode="AU" term="%22Kenet+G%22">Kenet G</searchLink>; The National Hemophilia Center and Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Hashomer, Tel Aviv University, Tel Aviv, Israel.<br /><searchLink fieldCode="AU" term="%22Lowe+G%22">Lowe G</searchLink>; West Midlands Adult Haemophilia Comprehensive Care Centre, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.<br /><searchLink fieldCode="AU" term="%22Key+NS%22">Key NS</searchLink>; UNC Blood Research Center, University of North Carolina, Chapel Hill, North Carolina, USA.<br /><searchLink fieldCode="AU" term="%22Millar+CM%22">Millar CM</searchLink>; Centre for Haematology, Imperial College London, London, UK.; Imperial College Healthcare NHS Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Pipe+SW%22">Pipe SW</searchLink>; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Chou+SC%22">Chou SC</searchLink>; Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Klamroth+R%22">Klamroth R</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Mason+J%22">Mason J</searchLink>; Queensland Haemophilia Centre, Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia.; University of Queensland, Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Chambost+H%22">Chambost H</searchLink>; AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France.<br /><searchLink fieldCode="AU" term="%22Peyvandi+F%22">Peyvandi F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Majerus+E%22">Majerus E</searchLink>; Department of Medicine, Washington University in St Louis, St Louis, Missouri, USA.<br /><searchLink fieldCode="AU" term="%22Pepperell+D%22">Pepperell D</searchLink>; Department of Haematology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia.<br /><searchLink fieldCode="AU" term="%22Tan+CW%22">Tan CW</searchLink>; Royal Adelaide Hospital, Adelaide, South Australia, Australia.<br /><searchLink fieldCode="AU" term="%22Yu+H%22">Yu H</searchLink>; BioMarin Pharmaceutical Inc, Novato, California, USA.<br /><searchLink fieldCode="AU" term="%22Chavele+KM%22">Chavele KM</searchLink>; BioMarin UK Ltd, London, UK.<br /><searchLink fieldCode="AU" term="%22Ozelo+MC%22">Ozelo MC</searchLink>; Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Sao Paulo, Brazil.
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  Data: <searchLink fieldCode="JN" term="%22101703775%22">Research and practice in thrombosis and haemostasis</searchLink> [Res Pract Thromb Haemost] 2026 Mar 27; Vol. 10 (3), pp. 103416. <i>Date of Electronic Publication: </i>2026 Mar 27 (<i>Print Publication: </i>2026).
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