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Substrate reduction theory: Glucosylceramide synthase inhibitors are therapeutic targets for lipid storing diseases. Glucosylceramide synthase inhibitors (EtDO-P4, C10 and Eliglustat tartrate) halt the synthesis of glucosylceramide, thereby preventing the downstream synthesis and accumulation of different sphingolipids involved in diseases such as Gaucher, Fabry’s, Tay-Sachs and GM1-Gangliosidosis.
