首个国产戈谢病酶替代疗法获批上市,价格有望比进口药低一半以上

第一财经
23 May

近日,北海康成(01228.HK)旗下罕见病药物注射用维拉苷酶β(商品名:戈芮宁)获批在中国上市,用于治疗12岁及以上青少年和成人I型和III型戈谢病患者,这是中国首款本土自主开发的戈谢病酶替代疗法药物。  戈谢病是一种罕见的遗传性溶酶体贮积症,患者临床表现包括肝脾肿大、贫血、骨痛和神经系统病变,严重时甚至可能因并发症危及生命。在中国,戈谢病已被纳入《第一批罕见病目录》。  大多数戈谢病患者分为I...

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