Ibiglustat L-Malic acid (Venglustat L-Malic acid) 是一种潜在的帕金森病、法布里病和戈谢病的治疗药物，是选择性的、葡糖神经酰胺合成酶 (GCS) 的变构抑制剂，能通过血脑屏障。
CAS：1629063-78-0
分子式：C24H30FN3O7S
分子量：523.57
纯度：98%
存储：Store at -20°C

Background:

Ibiglustat L-Malic acid (Venglustat L-Malic acid), a potential therapy for PD Parkinson’s disease, SRT in Fabry’s and Gaucher’s, is a selective, allosteric inhibitor of glucosylceramide synthase (GCS) with ability to cross the blood-brain barrier[1][2][3]. Glucosylceramide synthase[1].

Ibiglustat (SAR402671) (1 μM, 15 days) treated FD cells are close to the physiological level in untreated WT cells in GL-3 levels, suggesting that Ibiglustat can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes[4].

[1]. WO 2015089067 A1. [2]. Iva Stojkovska, et al. Molecular mechanisms of α-synuclein and GBA1 in Parkinson’s disease. Cell Tissue Res. 2017. [3]. Christoph Arenz, et al. Recent advances and novel treatments for sphingolipidoses. Future Med. Chem. (2017) 9(14), 1687-1700. [4]. Itier JM, et al. Effective clearance of GL-3 in a human iPSC-derived cardiomyocyte model of Fabry disease. J Inherit Metab Dis. 2014 Nov;37(6):1013-22.