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PHENO SMART
NCT06321965Recruiting
- Sponsor
- Hospices Civils de Lyon
- Start
- Jul 2024
- Primary completion
- Jan 2029
- Countries
- 1
01
Overview
Open-label, single-arm trial in spinal muscular atrophy (SMA), sponsored by Hospices Civils de Lyon. Primary endpoints: Markers of disease progression and description of different phenotypes at muscular and functional level at Month 24, Markers of disease progression and description of different phenotypes at fatigue level at Month 24 and 10 more. Started Jul 2024; status: recruiting.
02
Conditions
spinal muscular atrophy (SMA)
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