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对罕见疾病的药物重新定位:罕见疾病社区的进展和机遇

Anneliene Hechtelt Jonker1,2, Daniel O'Connor2,3, Maria Cavaller-Bellaubi2,4

  • 1Health Technology and Services Research Department, Technical Medical Centre, University of Twente, Enschede, Netherlands.

Frontiers in medicine
|February 1, 2024
PubMed
概括

药物再利用为罕见疾病提供了一个重要的策略. 关键的教训包括数据共享和早期监管参与,以及人工智能和患者参与等新兴趋势,以推进罕见疾病治疗.

关键词:
药物重用是为了改变药物的用途.创新 创新 创新 创新 创新 创新孤儿药物 孤儿药物是一种孤儿药物.罕见的疾病 罕见的疾病发展疗法 发展疗法没有满足的需要.

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