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].
In Vitro
Kinase Assay
Cell Assay
Animal Administration
References
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