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美国食品及药物管理局（FDA）宣布批准了第一款用于治疗小儿脊髓性肌萎缩症（SMA）的基因疗法Zolgensma。Zolgensma由AveXis公司研发，2018年4月，诺华收购了AveXis，并获得了该基因疗法。SMA是一种罕见的遗传性疾病，患儿用于编码运动神经元生存蛋白（SMN）的基因突变，因而出现进行性肌肉无力、瘫痪等症状，严重时，在2岁时死亡。

Zolgensma通过单次静脉注射，向患儿SMN基因的功能性拷贝，从而通过持续的SMN蛋白表达来阻止疾病进展。Zolgensma也成为FDA批准的首款用于治疗SMA的基因疗法。诺华将Zolgensma的售价定为212万美元，堪称‘史上最贵药物’。

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