1型戈谢病新药:Velaglucerase alfa
2010
戈谢病(Gaucher's disease)是一种罕见的遗传性疾病,其发病是由于编码溶酶体酶β-葡糖脑苷脂酶(β-glucocerebrosidase,GC)的基因发生突变所致,该酶可促进神经鞘脂葡糖脑苷脂的代谢,
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