FDA近日批准Celltech公司和Aetelion公司共同研制的Zavesca(miglustat) 胶囊治疗I型高歇病(Gaucher抯 disease),这是用于该适应证的第一个口服制剂。高歇病为一种罕见病,是葡萄糖脑苷脂缺陷所致的隐性遗传性代谢病,主要涉及肝、脾、淋巴结、肺及骨髓等单核巨噬细胞系统,全球约 The FDA recently approved the Zavesca (miglustat) capsule, a joint venture between Celltech and Aetelion, for the treatment of Gaucher’s disease, Type I, the first oral formulation to be used for this indication. Gaucher is a rare disease which is a recessive hereditary metabolic disease caused by glucocerebrosidase deficiency and mainly involves the mononuclear macrophage system such as liver, spleen, lymph nodes, lung and bone marrow,