Research Progress of Orphan Drugs and Discussion of Research and Development Power
10.3870/j.issn.1004-0781.2024.06.010
- VernacularTitle:孤儿药的研究进展与研发动力探讨
- Author:
Yang LI
1
;
Lei ZHU
Author Information
1. 中国医学科学院基础医学研究所,北京协和医学院基础学院药理系,北京 100005
- Keywords:
Orphan drugs;
Rare disease;
Drug research;
Development power
- From:
Herald of Medicine
2024;43(6):904-910
- CountryChina
- Language:Chinese
-
Abstract:
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.
