论文信息 - Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry - 字舞流文

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

D. D. De Vivo | K. Benguerba | Janbernd Kirschner | Kayoko Saito | Susana Quijano-Roy | Dheeraj Raju | Eric Faulkner | Nicole LaMarca | Perry B. Shieh | Eugenio Mercuri | Richard S. Finkel | J. Day | L. Servais | F. Muntoni | Crystal M. Proud | E. Tizzano | I. Desguerre | Rui Sun | Fred Anderson