从1994年 FGFR3靶点被证实与ACH相关,到2021年Vosoritide获批上市,ACH患者等待了近30年,才迎来首款针对性治疗药物。而随着Infigratinib叩响上市的大门,以及身后一众迭代疗法的逼近,ACH精准治疗的新大门正加速开启。这也是罕见病从被行业忽略到逐渐被重视的真实缩影。
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