基因疗法可引入功能基因来替代突变基因,以纠正或补偿缺陷和异常基因引起的疾病。 21世纪经济报道记者 魏笑 深圳报道 11月22日,美国FDA宣布澳大利亚制药公司CSL Behring的治疗B型血友病的一次性基因疗法Hemgenix获批,定价为350万美元,刷新了世界上最昂贵治疗方法的记录。 在不到半年的时间里,“全球最贵药物”已三易其主。今年8月,美国蓝鸟生物推出的用于治疗β-地中海贫血...
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