Migalastat is an orally active inhibitor of α-galactosidase A, functioning primarily as a molecular chaperone with an IC50 value of 0.04 μM for human α-Gal A. This compound selectively binds to the active site of specific unstable mutant forms of α-galactosidase A, promoting their proper transport to the lysosome. Upon dissociating in the acidic lysosomal environment, Migalastat enhances the biological activity of the mutant enzyme, making it a valuable tool in research related to lysosomal storage disorders.
Migalastat is an orally active inhibitor of α-galactosidase A, functioning primarily as a molecular chaperone with an IC50 value of 0.04 μM for human α-Gal A. This compound selectively binds to the active site of specific unstable mutant forms of α-galactosidase A, promoting their proper transport to the lysosome. Upon dissociating in the acidic lysosomal environment, Migalastat enhances the biological activity of the mutant enzyme, making it a valuable tool in research related to lysosomal storage disorders.
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