Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial
Valoctocogene roxaparvovec provides persistent FVIII expression, hemostatic control, and health-related quality of life improvements with no new safety signals.
Authors
- Nigel S. Key (ORCID: https://orcid.org/0000-0002-8930-4304)
- Robert Klamroth (ORCID: https://orcid.org/0000-0003-4194-8183)
- Steven W. Pipe (ORCID: https://orcid.org/0000-0003-2558-2089)
- Tara M. Robinson (ORCID: https://orcid.org/0000-0003-1513-707X)
- Gillian Lowe (ORCID: https://orcid.org/0000-0003-2527-9557)
- María Fernanda López Fernández (ORCID: https://orcid.org/0000-0003-2492-1843)
- Jane Mason (ORCID: https://orcid.org/0000-0002-8320-2884)
- Margareth C. Ozelo (ORCID: https://orcid.org/0000-0001-5938-0675)
- Gili Kenet (ORCID: https://orcid.org/0000-0003-3494-9823)
- Sheng‐Chieh Chou (ORCID: https://orcid.org/0000-0002-4121-019X)
- Hérvè Chambost (ORCID: https://orcid.org/0000-0002-5319-1969)
- Emily Symington (ORCID: https://orcid.org/0000-0003-2529-8168)
- Flora Peyvandi (ORCID: https://orcid.org/0000-0001-7423-9864)
- Dominic Pepperell (ORCID: https://orcid.org/0000-0002-5952-6395)
- Doris Quon (ORCID: https://orcid.org/0000-0002-9148-3268)
- Priyanka Raheja (ORCID: https://orcid.org/0000-0001-6365-2899)
- Christine Rivat
- Adam Giermasz
- Carolyn M. Millar
- Elaine M. Majerus
- Johnny Ndoni Mahlangu (ORCID: https://orcid.org/0000-0001-5781-7669)
- Hua Yu (ORCID: https://orcid.org/0000-0002-7255-485X)
- Andrew D. Leavitt (ORCID: https://orcid.org/0000-0003-4827-0790)
- Bella Madan (ORCID: https://orcid.org/0000-0003-4858-4888)
Institutions
- National Health Laboratory Service (ZA)
- University of North Carolina at Chapel Hill (US)
- National Health Service (GB)
- University Hospitals Birmingham NHS Foundation Trust (GB)
- University of Bonn (DE)
- Imperial College Healthcare NHS Trust (GB)
- Tel Aviv University (IL)
- The University of Queensland (AU)
- University of California, San Francisco (US)
- Universidade Estadual de Campinas (UNICAMP) (BR)
- University of Milan (IT)
- Guy's and St Thomas' NHS Foundation Trust (GB)
- Washington University in St. Louis (US)
- Barts Health NHS Trust (GB)
- University of Michigan (US)
- University Hospital Bonn (DE)
- Sheba Medical Center (IL)
- Fiona Stanley Hospital (AU)
- Cambridge University Hospitals NHS Foundation Trust (GB)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Assistance Publique Hôpitaux de Marseille (FR)
- National Taiwan University Hospital (TW)
- Complexo Hospitalario Universitario A Coruña (ES)
- Unité de recherche sur les maladies cardiovasculaires et métaboliques (FR)
- Orthopaedic Institute for Children (US)
- Klinikum im Friedrichshain (DE)
- Cancer Care Services (US)
- Imperial College London (GB)
- University of California, Davis (US)
- BioMarin (United States) (US)
Publication Details
- Journal
- Research and Practice in Thrombosis and Haemostasis
- Published
- 2024-10-31
- DOI
- https://doi.org/10.1016/j.rpth.2024.102615
- Citations
- 29
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 13.18
Funders
- BioMarin Pharmaceutical