Migalastat
Galactosidase inhibitor; pharmacological chaperone / Migalastat is a potent, selective, and orally available inhibitor of α-galactosidase A (IC50= 40 nM).1 It binds to mutant misfolded α-GalA shifting the folding behavior toward functional active conformation (pharmacological chaperone activity) followed by trafficking to the lysosomes.2 Migalastat is active broadly across species from Drosophila3 to human4. Clinically useful agent for Fabry disease.5
Biochemicals & reagents
75172-81-5
1-deoxygalactonojirimycin; DDIG; AT1001; DGJ; GR181413
1 Asano et al. (2000), In vitro inhibition and intracellular enhancement of lysosomal α-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives; Eur. J. Biochem. 267 4179 2 Siekierska et al. (2012), α-Galactosidase aggregation is a determinant of pharmacological chaperone efficacy on Fabry disease mutants; J. Biol. Chem. 287 28386 3 Braunstein et al. (2020), Misfolding of Lysosomal α-Galactosidase a in a Fly Model and Its Alleviation by the Pharmacological Chaperone Migalastat; Int. J. Mol. Sci. 21 7397 4 Benjamin et al. (2009), The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines; J. Inherit. Metab. Dis. 32 424 5 McCafferty and Scott (2019), Migalastat: A Review in Fabry Disease; Drugs 79 543
-20°C
TARGET: Galactosidase; Chaperone -- PATHWAY: Intracellular transport