Post-marketing specified use-results survey of Zolgensma intrathecal injection in spinal muscular atrophy

Open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoints: Incidence and timing of serious adverse events and all adverse drug reactions and Incidence and timing of predefined safety specification events. Started Oct 2026; status: recruiting.