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Updated: Jul 6, 2025

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药理学研究在孤儿药物开发中的实施情况

Saki Yokoshiki1,2, Teruyo Arato3,4

  • 1Graduate School of Medicine, Hokkaido University, Kita 15, Nishi 7, Kita-ku, Sapporo, 060-8638, Japan.

Orphanet journal of rare diseases
|January 3, 2024
PubMed
概括

由于未知的疾病机制和缺乏动物模型,开发孤儿药物具有挑战性. 这项研究审查了日本的孤儿药物批准,以找到有效的非临床疗效评估方法,表明灵活的研究设计可以帮助批准.

关键词:
动物模型动物模型生物标志物 生物标志物细胞系 细胞系 细胞系指导指导的指导.非临床研究是指非临床研究.孤儿药物 孤儿药物 是一种孤儿药物.罕见疾病是一种罕见的疾病.

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