Ibiglustat CAS NO.1401090-53-6

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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].