Eliglustat, also known as Genz-112638, is is a potent and selective glucosylceramide synthase inhibitor. Eliglustat was approved for Gaucher's disease. Commonly used as the tartrate salt, the compound is believed to work by inhibition of glucosylceramide synthase. According to an article in Journal of the American Medical Association the oral substrate reduction therapy resulted in "significant improvements in spleen volume, hemoglobin level, liver volume, and platelet count" in untreated adults with Gaucher disease Type 1.
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