关键词: Drug repurposing Innovation Orphan drugs Rare diseases Therapy development Unmet need

Mesh : Rare Diseases / drug therapy Humans Drug Repositioning

来  源:   DOI:10.1016/bs.pmbts.2024.03.034

Abstract:
Repurposing drugs for rare diseases is a creative and cost-efficient method for creating new treatment options for certain conditions. This technique entails repurposing existing pharmaceuticals for new uses by utilizing established information regarding pharmacological characteristics, modes of operation, safety profiles, and interactions with biological systems. Creating new treatments for uncommon diseases is frequently difficult because of factors including small patient groups, disease intricacy, and insufficient knowledge of disease pathobiology. Drug repurposing is a more efficient and cost-effective approach compared to developing new drugs from scratch. It typically requires collaboration among academia, pharmaceutical firms, and patient advocacy groups.
摘要:
为罕见疾病重新使用药物是一种创造性且具有成本效益的方法,可以为某些疾病创造新的治疗选择。该技术需要通过利用有关药理学特征的既定信息,将现有药物重新用于新用途。操作模式,安全概况,以及与生物系统的相互作用。由于包括小患者群体在内的因素,为罕见疾病创造新的治疗方法通常很困难。疾病错综复杂,和疾病病理生物学知识不足。与从头开始开发新药物相比,药物再利用是一种更有效和更具成本效益的方法。它通常需要学术界之间的合作,制药公司,和患者倡导团体。
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