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Migalastat in Pediatric Fabry Disease (2 to <12 Years)
2025-521244-38-00Phase 3Authorised
- Sponsor
- Amicus Therapeutics
- Start
- Dec 2025
- Modality
- Classical Binding Small Molecules → Small Molecules
- Countries
- 5
01
Overview
Phase 3, open-label, single-arm trial of migalastat in Fabry disease (FD), sponsored by Amicus Therapeutics. Primary endpoints: Pharmacokinetics of migalastat, Safety - adverse events and 7 more. Planned start Dec 2025; status: authorised.
02
Drugs
migalastat
03
Conditions
Fabry disease (FD)
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