Migalastat Hydrochloride

CAS 1185306-07-0 7 FDA-DMF suppliersSmall Molecule

Overview

Migalastat Hydrochloride is an orally administered pharmacological chaperone designed to treat Fabry disease by stabilizing specific mutant forms of the alpha-Galactosidase A enzyme. It aids in trafficking the dysfunctional enzyme to lysosomes, thus potentially restoring its normal function.

Therapeutic areas

Genetic DisordersRare DiseasesMetabolic Disorders

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