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Miglustat (Zavesca) versus placebo in children with mucopolysaccharidosis type III
2006-004661-34Phase 2Completed
- Sponsor
- Hospices Civils de Lyon
- Start
- Oct 2006
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2 trial of miglustat in Sanfilippo syndrome (2 arms), sponsored by Hospices Civils de Lyon. Primary endpoint: Adaptive behavior stabilization or improvement at 6 months. Started Oct 2006; status: completed.
02
Drugs
miglustat
03
Conditions
Sanfilippo syndrome
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