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售价212万美元,FDA批准“史上最贵药物”

 4 years ago
source link: https://tech.sina.com.cn/d/f/2019-05-27/doc-ihvhiews4859558.shtml
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来源:环球科学5月24日,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

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