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Onasemnogene abeparvovec in Spinal Muscular Atrophy Type 1
NCT03837184Phase 3Completed
- Sponsor
- Novartis
- Start
- May 2019
- Primary completion
- Jun 2021
- Countries
- 3
01
Overview
Phase 3, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy type 1 (SMA1), sponsored by Novartis. Primary endpoint: Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds. Started May 2019; status: completed.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy type 1 (SMA1)
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