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Intrathecal OAV101 versus sham in later-onset type 2 spinal muscular atrophy
2021-003474-31Phase 3Completed
- Sponsor
- Novartis
- Start
- Nov 2021
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of onasemnogene abeparvovec in spinal muscular atrophy type 2 (SMA2) (2 arms), sponsored by Novartis. Primary endpoint: Change from baseline in Hammersmith Functional Motor Scale-Expanded total score. Started Nov 2021; status: completed.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy type 2 (SMA2)
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