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