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Onasemnogene abeparvovec-xioi in pre-symptomatic spinal muscular atrophy
NCT03505099Phase 3Completed
- Sponsor
- Novartis
- Start
- Apr 2018
- Primary completion
- Jun 2021
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 6
01
Overview
A Phase 3 trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Started Apr 2018; status: completed.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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