Nat Rev Drug Discov. 2020 Feb;19(2):77-78. doi: 10.1038/d41573-019-00180-y.
A lack of robust knowledge of the number of rare diseases and the number of people affected by them limits the development of approaches to ameliorate the substantial cumulative burden of rare diseases. Here, we call for coordinated efforts to more precisely define rare diseases.
缺乏对罕见病数量和受影响人数的充分了解,限制了改善罕见病巨大累积负担的方法的发展。在这里,我们呼吁协调努力,以更准确地定义罕见病。