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Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with OAV101
2024-511707-42-00Phase 3Authorised
- Sponsor
- Novartis
- Start
- Aug 2024
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Countries
- 22
01
Overview
Phase 3, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoints: Treatment-emergent serious adverse events and Treatment-emergent adverse events of special interest. Planned start Aug 2024; status: authorised.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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