摘要
罕见病发病率极低,但种类繁多,且大部分疾病病情严重,预后较差,孤儿药缺乏一直是罕见病治疗领域的瓶颈问题。随着基因工程、大数据分析和人工智能等技术手段的进步,基因治疗、抗体疗法、酶替代疗法、药物再利用等已逐步成为孤儿药研发的重点方向。虽然我国罕见病研究起步较晚,但近年来日益受到重视,一系列举措鼓励医药企业积极参与孤儿药研发,并取得快速进步。该文分析孤儿药的研究进展,并探讨开发孤儿药的动力,希望能够助力我国孤儿药的未来研发。
The incidence of rare diseases is extremely low.But there are many kinds of rare diseases and most of them are serious and have poor prognosis.The lack of orphan drugs has always been a bottleneck in the field of rare disease treatment.Gene therapy,antibody therapy,enzyme replacement therapy and drug repurposing have gradually become key directions of orphan drug research and development with the advancement of genetic engineering,big data analysis and artificial intelligence.Although the research on rare diseases started late in China,it has received more and more attention in recent years.A series of measures have been taken to encourage pharmaceutical enterprises to actively participate in the research and development of orphan drugs,and have achieved rapid progress.This article analyzes the research progress of orphan drugs,and explores the driving force of developing orphan drugs,aiming to contribute to the future research and development of orphan drugs in China.
作者
李阳
朱蕾
LI Yang;ZHU Lei(Department of Pharmacology,Institute of Basic Medical Sciences,Chinese Academy of Medical Sciences and School of Basic Medicine Peking Union Medical College,Beijing 100005,China)
出处
《医药导报》
CAS
北大核心
2024年第6期904-910,共7页
Herald of Medicine
基金
中国医学科学院医学与健康科技创新工程(2021-I2M-1-005)
北京协和医院中央高水平医院临床科研专项(2022-PUMCH-C-025)。
关键词
孤儿药
罕见病
药物研发
研发动力
Orphan drugs
Rare disease
Drug research
Development power
