Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

NCT03505099 · clinicaltrials.gov ↗
PHASE3
Phase
COMPLETED
Status
30
Enrollment
INDUSTRY
Sponsor class

Conditions

Interventions

Sponsor

Novartis Gene Therapies

Collaborators