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18 小时
FDA批准首款CT疾病新药:拯救罕见病患者的希望
近日,值得关注的医学进展传来好消息:美国食品药品监督管理局(FDA)成功批准了Mirum Pharmaceuticals旗下的Ctexli(chenodiol),这标志着首款针对成人脑腱黄瘤病(CTX) 的药物获得上市许可。CTX是一种罕见且进行性的遗传性胆固醇代谢障碍,它可能损害身体的多种功能,成为许多患者生活中的沉重负担。
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