Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry
使用 Onasemnogene Abeparvovec 单药治疗脊髓性肌萎缩症患者的真实世界结果：来自 RESTORE 注册研究的发现
期刊:Journal of Neuromuscular Diseases
影响因子:3.4
doi:10.3233/JND-230122
Servais, Laurent; Day, John W; De Vivo, Darryl C; Kirschner, Janbernd; Mercuri, Eugenio; Muntoni, Francesco; Proud, Crystal M; Shieh, Perry B; Tizzano, Eduardo F; Quijano-Roy, Susana; Desguerre, Isabelle; Saito, Kayoko; Faulkner, Eric; Benguerba, Kamal M; Raju, Dheeraj; LaMarca, Nicole; Sun, Rui; Anderson, Frederick A; Finkel, Richard S
