Miglustat HCl

Orally active ceramide-specific glycosyltransferase and α-glucosidase I and II inhibitor.1 Rescues trafficking-deficient F508del-CFTR in human airway epithelial cells via inhibition of ER α-glucosidases I and II.2 Pharmacological chaperone for glucocerebrosidase degradation.3 Clinically useful agent for Gaucher disease type 1.4 Stabilizes neurological disorders in Niemann-Pick disease type C.5