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Phase IIIb open-label single-arm study of intrathecal OAV101 in previously treated pediatric SMA
NL-OMON56088Phase 3Completed
- Sponsor
- Novartis
- Start
- Jan 2023
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoint: Safety and tolerability over 52 weeks. Started Jan 2023; status: completed.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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