Ibiglustat L-Malic acid (Synonyms: Venglustat (L-Malic acid); SAR402671 (L-Malic acid); GZ402671 (L-Malic acid)) |
| رقم الكتالوجGC36288 |
Ibiglustat (Venglustat) L-Malic acid هو مثبط للجلوكوزيل سيراميد سينسيز (GCS) النشط عن طريق الفم والذي يخترق الدماغ.
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Cas No.: 1629063-78-0
Sample solution is provided at 25 µL, 10mM.
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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.
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