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Open-label 12‑month migalastat study in pediatric Fabry disease with amenable GLA variants
2017-000146-21Phase 3Unknown
- Sponsor
- Amicus Therapeutics
- Start
- Mar 2019
- Modality
- Classical Binding Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of migalastat in Fabry disease (FD), sponsored by Amicus Therapeutics. Primary endpoint: Incidence of treatment-emergent adverse events, serious adverse events, and adverse events leading to discontinuation. Status: unknown.
02
Drugs
migalastat
03
Conditions
Fabry disease (FD)
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