IbigluSTAT 是一种可透过血脑屏障的、具有口服活性的葡萄糖神经酰胺合成酶(GCS)抑制剂。它可用于戈谢病 3 型、法布瑞氏症、与 GBA 突变相关的帕金森病、GM2 神经节苷脂病和常染色体显性多囊肾病的研究。

Ibiglustat is a potent and selective Glucosylceramide synthase inhibitor and ceramide glucosyltransferase inhibitor. Ibiglustat blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3. Ibiglustat is potentially useful for treating Fabry disease. Fabry disease is a rare lysosomal storage disorder, which results in abnormal tissue deposits of a particular fatty substance called globotriaosylceramide (GL-3 or Gb3) throughout the body.

Ibiglustat is in phase II Clinical trials for the treatment of Fabry's disease. Fabry disease, an X-linked disorder of glycosphingolipids that is caused by mutations of the GLA gene that codes for α-galactosidase A, leads to dysfunction of many cell types and includes a systemic vasculopathy [1].

1401090-53-6

C20H24FN3O2S

389.49

GZ402671;SAR402671;Venglustat

DMSO:10 mM
Powder: -20°C for 3 years
In solvent: -80°C for 2 years