Eliglustat hemitartrate是一种高效的、特异性的、有口服活性的葡糖脑苷脂合成酶抑制剂(IC50:24 nM)。

Eliglustat Hemitartrate, with an IC50 of 24 nM, is a specific, potent, oral active glucocerebral glycosidase inhibitor.

Eliglustat tartrate shows good potency with an IC50 of 24 nM and specificity against the target enzyme.

A novel inhibitor of glucosylceramide synthase (Genz-112638) and assessed its activity in a murine model of Gaucher disease (D409V/null).?Biochemical characterization of Genz-112638 showed good potency (IC(50) approximately 24nM) and specificity against the target enzyme.?Mice that received drug prior to significant accumulation of substrate (10 weeks of age) showed reduced levels of glucosylceramide and number of Gaucher cells in the spleen, lung and liver when compared to age-matched control animals.?Treatment of older mice that already displayed significant amounts of tissue glucosylceramide (7 months old) resulted in arrest of further accumulation of the substrate and appearance of additional Gaucher cells in affected organs.?These data indicate that substrate inhibition therapy with Genz-112638 represents a viable alternate approach to enzyme therapy to treat the visceral pathology in Gaucher disease.

928659-70-5

C27H42N2O10

554.63

依利格鲁司特酒石酸盐;Genz-112638;Eliglustat tartrate;Eliglustat hemitartrate

DMSO:50 mg/mL (52.13 mM)
H2O:50 mg/mL (52.13 mM)
Powder: -20°C for 3 years
In solvent: -80°C for 2 years