中国首款针对渐冻症的精准治疗药物获批上市了

美港电讯
08 Oct 2024

【中国首款针对渐冻症的精准治疗药物获批上市了】金十数据10月8日讯,渤健中国宣布,中国药监局附条件批准托夫生注射液(商品名:凯盛迪)用于治疗携带超氧化物歧化酶1(SOD1)基因突变的ALS成人患者,这也成为中国首个获批上市的ALS精准治疗药物。此次获批上市的托夫生注射液,是一种反义寡核苷酸(ASO)药物,可通过减少SOD1蛋白合成,减少毒性SOD1蛋白的蓄积,从而减轻运动神经元的损伤,减缓疾病进展。2023年4月,该药率先在美国获批上市,也是目前全球唯一一款针对SOD1-ALS的对因治疗药物。

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