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SMART02 valproic acid in childhood-onset spinal muscular atrophy
jRCT2091220231Completed
- Sponsor
- Institute of Medical Genetics, Tokyo Women's Medical University
- Start
- Dec 2015
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Randomised trial in spinal muscular atrophy (SMA) comparing Valproic acid (SMART) arm with Placebo arm (2 arms). Primary endpoint: HFMSE score. Started Dec 2015; status: completed.
02
Drugs
valproic acid
03
Conditions
spinal muscular atrophy (SMA)
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