Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Status:
Completed
Trial end date:
2021-02-02
Target enrollment:
Participant gender:
Summary
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 variants.