Eliglustat tartrate (GENZ-112638; Genz-99067; Cerdelga), the tartrate salt of eliglustat, is a specific and orally bioactive glucocerebroside synthase inhibitor (IC50 = 24 nM) that has been approved by the FDA in August 2014 for the treatment for Gaucher's disease type 1 (GD1). It is 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.

纯度：≥98%

CAS：928659-70-5