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Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with OAV101
jRCT2033230642Recruiting
- Sponsor
- Novartis
- Start
- Mar 2024
- Modality
- Gene Therapies
01
Overview
Open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoint: Treatment-emergent serious adverse events. Started Mar 2024; status: recruiting.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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