Ibiglustat

一种葡萄糖神经酰胺合成酶抑制剂；在法布里病（Fabry disease）的Gla基因敲除（Gla-/-）小鼠模型中，减少多种组织中Globotriaosylceramide（Gb3）和溶酶体Globotriaosylceramide（lyso-Gb3）的积累；在相同模型中，减少热板试验中爪子回缩的潜伏期；在神经源性高雪病（neuronopathic Gaucher disease）的转基因4L;C*小鼠模型中，通过饮食摄入量为0.03%（重量比）时，减少胶质细胞增生，延缓共济失调的发作，并延长寿命。A GlcCer synthase inhibitor; reduces Gb3 and lyso-Gb3 accumulation in a variety of tissues in the Gla-/- mouse model of Fabry disease; reduces the latency to paw withdrawal in the hot plate test in the same model; reduces gliosis, delays the onset of ataxia, and increases lifespan in the transgenic 4L;C* mouse model of neuronopathic Gaucher disease at 0.03% w/w in the diet.分子式C20H24FN3O2S分子量389.5CC(C)(NC(O[C@@H]1CN2CCC1CC2)=O)C3=CSC(C4=CC=C(F)C=C4)=N3