Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial
在 GENEr8-1 3 期试验中,接受 valoctocogene roxaparvovec 基因转移治疗重度 A 型血友病 4 年后的疗效、安全性和生活质量
期刊:Research and Practice in Thrombosis and Haemostasis
影响因子:3.4
doi:10.1016/j.rpth.2024.102615
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; Madan, Bella; Chou, Sheng-Chieh; Klamroth, Robert; Mason, Jane; Chambost, Hervé; Peyvandi, Flora; Majerus, Elaine; Pepperell, Dominic; Rivat, Christine; Yu, Hua; Robinson, Tara M; Ozelo, Margareth C