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STREAM: Real-world Safety & Effectiveness of Intrathecal Onasemnogene Abeparvovec in SMA
NCT07448610Phase 4Recruiting
- Sponsor
- Novartis
- Start
- Aug 2026
- Primary completion
- May 2032
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
A Phase 4 trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Started Aug 2026; status: recruiting.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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