Lucerastat,141206-42-0

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Lucerastat

CAS No. : 141206-42-0

MCE 国际站：Lucerastat

产品活性：Lucerasta 是Miglustat 的半乳糖形式，是具有口服活性的葡萄糖酰神经酰胺合成酶 (GCS) 的抑制剂。Lucerastat 有用于Fabry 疾病研究的潜能。

研究领域：Neuronal Signaling

作用靶点：Glucosylceramide Synthase (GCS)

In Vitro: Fabry patient-derived fibroblasts with the genotypes R301G (residual -GalA activity; 20%) R220X (<3%) and W162X (<1%).

In Vivo: Lucerastat (1200 mg/kg/day food admix), a GCS inhibitor, reduces Gb3 in the absence of residual-GalA activity.

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