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SMART01 valproic acid in childhood-onset spinal muscular atrophy
jRCT2091220190Completed
- Sponsor
- Institute of Medical Genetics, Tokyo Women's Medical University
- Start
- Aug 2014
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label, single-arm trial of valproic acid in spinal muscular atrophy (SMA), sponsored by Institute of Medical Genetics, Tokyo Women's Medical University. Primary endpoint: SMN protein level by imaging flow cytometry. Started Aug 2014; status: completed.
02
Drugs
valproic acid
03
Conditions
spinal muscular atrophy (SMA)
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