纯度:>98% by TLC,;NMR (Conforms)
分子式:C6H13NO4 ? HCl
分子量:199.63
溶剂:DMSO (50 mg/ml)
性状:White solid
存储:-20°C
Activity (short version):Galactosidase inhibitor; pharmacological chaperone
Function / Pharmacology: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