Rare diseases continue to pose a formidable public health challenge with lack or absence of effective treatments,demonstrating an immense need for rare disease drugs.However,the development of rare disease drugs remai...Rare diseases continue to pose a formidable public health challenge with lack or absence of effective treatments,demonstrating an immense need for rare disease drugs.However,the development of rare disease drugs remains uneven globally due to disparities in resource allocation,policy support,and medical infrastructure[1].Compared with other regions such as the USA and the EU,China exhibits distinct needs related to its particular disease spectrum,incidence,prevalence,genetic backgrounds,aetiology,and clinical practices[2].展开更多
基金supported by the National Key Research and Development Program of China(2023YFC2508500)Beijing Municipal Health Commission(Beijing Demonstration Research Ward BCRW20200303)+1 种基金the National Natural Science Foundation of China(82272951 and 82272953)the Chinese Academy of Medical Sciences(2022-I2M-C&T-B-070).
文摘Rare diseases continue to pose a formidable public health challenge with lack or absence of effective treatments,demonstrating an immense need for rare disease drugs.However,the development of rare disease drugs remains uneven globally due to disparities in resource allocation,policy support,and medical infrastructure[1].Compared with other regions such as the USA and the EU,China exhibits distinct needs related to its particular disease spectrum,incidence,prevalence,genetic backgrounds,aetiology,and clinical practices[2].
