LAUSR

In 2018,National Science Review interviewed Prof. David R. Liu of the Broad Institute, who pioneered CRISPR-based genomeediting technologies [1]. As soon as this interview was posted on China’s social media, a father of a Duchenne muscular dystrophy (DMD) patient contacted us, hoping Liu’s innovation could save his son. The DMD patients usually lose the ability to stand and walk by the age of 12, and many do not survive beyond 20 years old. Similarly to rare-disease families throughout the world, rare-disease patients in China are desperately in search of possible therapy. Since 2015, attention on rare diseases has been mounting in China, with emerging new policies and programs. Compared with their counterparts in the USA and Europe, rare-disease patients in China are facing many more challenges in receiving diagnosis and treatment. On 18–19 December 2021, the 2021ChinaConference onRareDiseases was heldonline.Hundredsof physicians, researchers, policy-makers, patients and medical-industry representatives gathered to discuss multiple aspects of rare diseases in China.