Last updated on September 2018

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Mutations


Brief description of study

This is an open-label study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects 12 to <18 years of age with Fabry disease and amenable GLA mutations.

Clinical Study Identifier: TX212279

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Emma Gonzalez

University of South Florida-Morsani College of Medicine
Tampa, FL USA
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Recruitment Status: Open


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