Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat succinate can be used for the research of Gaucher disease type 3, Parkinsons disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease.
性状
Solid
体外研究(In Vitro)
Ibiglustat (SAR402671) succinate (1 μM, 15 days; Fabry disease (FD) cells) is close to the physiological level in untreated WT cells in GL-3 levels, suggesting that Ibiglustat succinate can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes. has not independently confirmed the accuracy of these methods. They are for reference only.
运输条件
Room temperature in continental US; may vary elsewhere.
储存方式
Powder -20°C 3 years;4°C 2 years
ClinicalTrial
参考文献
[1]. Viel C, et al. Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model. Sci Rep. 2021;11(1):20945. Published 2021 Oct 22. [2]. Peterschmitt MJ, et al. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021;10(1):86-98.