罕见病巨头Ultragenyx(RARE.US)反义寡核苷酸疗法挺进3期临床剑指天使综合征

智通财经04-16

智通财经APP获悉，Ultragenyx Pharmaceutical(RARE.US)宣布其用于治疗天使综合征(Angelman syndrome)的在研疗法GTX-102在1/2期临床试验中获得的最新数据。数据分析显示，接受GTX-102治疗的扩展队列A和B的患者，在第170天显示出快速且具有临床意义的多领域功能改善。此外，部分剂量递增队列患者在第758天显示出长期和持续的临床益处，远超过自然...

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罕见病巨头Ultragenyx(RARE.US)反义寡核苷酸疗法挺进3期临床 剑指天使综合征

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罕见病巨头Ultragenyx(RARE.US)反义寡核苷酸疗法挺进3期临床剑指天使综合征