Ibiglustat (Synonyms: Genz-682452, GZ/SAR402671, Venglustat) |
| رقم الكتالوجGC19196 |
Ibiglustat (Venglustat) هو مثبط نشط للجلوكوزيلسيراميد سينسيز (GCS) عن طريق الفم ، يخترق الدماغ
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Cas No.: 1401090-53-6
Sample solution is provided at 25 µL, 10mM.
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Ibiglustat is an inhibitor of glucosylceramide synthase extracted from patent WO 2015089067 A1, compound example 64.
Ibiglustat is in phase II clinical trials for the treatment of Fabry's disease. Fabry disease, an X-linked disorder of glycosphingolipids that is caused by mutations of the GLA gene that codes for α-galactosidase A, leads to dysfunction of many cell types and includes a systemic vasculopathy[2]. Substrate reduction therapy is an approach that reduces the synthesis of lipids reaching the lysosome through inhibition of glucosylceramide synthase (GCS)[1].
References:
[1]. WO 2015089067 A1
[2]. Schiffmann R, et al. Fabry disease. Handb Clin Neurol. 2015;132:231-48.
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