Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial - Leavitt, Andrew D.; Mahlangu, Johnny; Raheja, Priyanka; Symington, Emily; Quon, Doris V.; Giermasz, Adam; López Fernández, Maria Fernanda; Kenet, Gili; Lowe, Gillian; Key, Nigel S.; Millar, Carolyn M.; Pipe, Steven W.; et al. (Research and practice in thrombosis and haemostasis, 2024)
Keywords: adeno-associated virus; clinical trial; gene therapy; hemophilia A; Life Sciences; Original; quality of life; clinical trial; hemophilia A; gene therapy; adeno-associated virus; quality of life