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Long-term follow-up of patients with spinal muscular atrophy treated with OAV101
2021-006781-21Phase 3Active
- Sponsor
- Novartis
- Start
- Jul 2022
- Modality
- Gene Therapies
- Countries
- 1
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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. Started Jul 2022; status: active.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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