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Nature Medicine|December 8, 2025
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trialJennifer M Kwon, Francina Munell, Laure Le Goff, et al.
Annals of Neurology|August 4, 2023
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR)Craig M Zaidman, Crystal M Proud, Craig M McDonald, et al.
Journal of Neuromuscular Diseases|January 22, 2024
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE RegistryLaurent Servais, John W Day, Darryl C De Vivo, et al.
Nature Medicine|December 8, 2025
Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trialCrystal M Proud, Dũng Chí Vũ, Jo M Wilmshurst, et al.
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