Synthesis and Analytic Method of N-Butyldeoxyjirinomycin

来源 :第九届IUPAC化学生物学国际研讨会暨第八届世界华人药物化学研讨会 | 被引量 : 0次 | 上传用户:erapple
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  Type Ⅰ Gaucher disease is an inherited disease caused by an abnormal accumulation of glucosylceramide in bone marrow cells and visceral organs due to the deficiency of glucocerebrosidase.Currently,the common treatments of type Ⅰ Gaucher disease are enzyme replacement therapy or bone marrow transplant,which are both time consuming and expensive.Although N-butyldeoxyjirinomycin (miglustat) is approved by the US Food and Drug Administration for the treatment of Gaucher disease,still,the expensive cost has made it difficult for the patients to access.
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