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Long-term follow-up of OAV101-treated patients with spinal muscular atrophy
NL-OMON56701Phase 3Not yet recruiting
- Sponsor
- Novartis
- Start
- May 2023
- Modality
- Gene Therapies
- Countries
- 1
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Overview
Phase 3, open-label trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA) (2 arms), sponsored by Novartis. Primary endpoints: Treatment-emergent serious adverse events and Treatment-emergent adverse events of special interest. Planned start May 2023; status: not yet recruiting.
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Drugs
onasemnogene abeparvovec
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Conditions
spinal muscular atrophy (SMA)
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