Migalastat hydrochloride-15N is a stable isotope-labeled form of Migalastat hydrochloride, an orally active molecular chaperone targeting α-galactosidase A. It exhibits a potent IC50 of 0.04 μM for human α-Gal A, facilitating the proper folding and lysosomal transport of certain unstable mutant forms of the enzyme. Upon dissociation in the acidic lysosomal environment, Migalastat promotes restoration of biological activity, making it valuable for research in lysosomal storage disorders and enzyme replacement therapies.
Migalastat hydrochloride-15N is a stable isotope-labeled form of Migalastat hydrochloride, an orally active molecular chaperone targeting α-galactosidase A. It exhibits a potent IC50 of 0.04 μM for human α-Gal A, facilitating the proper folding and lysosomal transport of certain unstable mutant forms of the enzyme. Upon dissociation in the acidic lysosomal environment, Migalastat promotes restoration of biological activity, making it valuable for research in lysosomal storage disorders and enzyme replacement therapies.
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