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Miglustat for infantile Tay-Sachs and Sandhoff disease
IRCT20181023041435N1Phase 4Completed
- Sponsor
- Tehran University of Medical Sciences
- Start
- Nov 2018
- Modality
- Small Molecules
- Countries
- 1
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Overview
Phase 4, open-label, single-arm trial of miglustat in Tay-Sachs disease (TSD) and Sandhoff disease, sponsored by Tehran University of Medical Sciences. Primary endpoints: Hospitalization, Feeding route and 4 more. Started Nov 2018; status: completed.
02
Drugs
miglustat
03
Conditions
Tay-Sachs disease (TSD)Sandhoff disease
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