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Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with AVXS-101
jRCT1080225209Phase 3Completed
- Sponsor
- Novartis
- Start
- May 2020
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
01
Overview
Phase 3, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Started May 2020; status: completed.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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