Migalastat (GR181413A free base) is an orally active α-galactosidase A molecular chaperone, with an IC 50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity.
Products | Products > Signaling Pathways > Metabolic Enzyme/Protease | Products > Research Areas > Others | Products > Signaling Pathways > Glycosidase
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