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eliglustat
Eliglustat (INN, USAN;〔 By subscription only〕 trade name Cerdelga) is a treatment for Gaucher's disease developed by Genzyme Corp that was approved by the FDA August 2014. 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.
==Cost==
In 2014, the annual cost of Cerdelga hard gelatin capsules taken orally twice a day was $310,250. Genzyme’s flagship Imiglucerase (brand name Cerezyme) cost about $300,000 for the infusions if taken twice a month. Manufacturing costs for Cerdelga are slightly lower than for Cerezyme. Genzyme's maintains higher prices for orphan drugs—most often paid for by insurers— in order to remain financially sustainable.〔
抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』
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