Onasemnogene abeparvovec Completed Phase 3 Trials for Spinal Muscular Atrophy (SMA) Treatment

IndicationsStatusPurposePhase
CompletedTreatment3
clinicaltrials.gov IdentifierTitleDrugs
NCT03505099Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2