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Chaperone Therapy
Migalastat HCl 150 mg for Fabry Disease
Recruiting2 awardsPhase 3
Pittsburgh, Pennsylvania
This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to \<18 years of age with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.
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