957230-65-8

Off-White Solid

Used as human acid ?glucosidase inhibitor and for the treatment of Gaucher disease

Used as human acid β-glucosidase inhibitor and for the treatment of Gaucher disease

Isofagomine tartrate (957230-65-8) is a competitive inhibitor of human lysosomal β-glucosidase, IC50?= 0.06 μM.1?Increases the activity of N370S mutant acid β-glucosidase (GlcCerase) in Gaucher fibroblasts.2?Displays a direct pharmacological chaperone effect greatly increasing the amount of newly synthesized GlcCerase that traffics out of the ER.2

1) Kuriyama?et al.?(2008),?In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell culture; Bioorg. Med. Chem.,?16?7330 2) Steet?et al. (2006),?The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanisms; Proc. Natl. Acad. Sci. USA,?103?13813