Clinical Trial Update: Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease [Phase 3]

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Summary

A Phase 3 trial evaluating migalastat in pediatric patients over 12 years with Fabry disease has completed, reporting safety, pharmacodynamics, and efficacy res…

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Diseases / indications

• Fabry Disease

Drugs mentioned

• Migalastat (migalastat)

Snippet

Sponsor: Amicus Therapeutics | Conditions: Fabry Disease | Phase 3 | Status: COMPLETED | Results posted: 2025-06-17