Migalastat is a potent, selective, and orally available inhibitor of alpha-galactosidase A (IC50= 40 nM).1 It binds to mutant misfolded alpha-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
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