Comprehensive analysis of 2097 patients with dystrophinopathy based on a database from 2011 to 2021.

Lei ZhaoYiyun ShiChaoping HuShuizhen ZhouHui LiLifeng ZhangChuang QianYiyao ZhouYi WangXihua Li

Published in: Orphanet journal of rare diseases (2024)

This is one of the largest studies to have evaluated the natural history of dystrophinopathy in China, which is particularly conducive to the recruitment of eligible patients for clinical trials and the provision of real-world data to support drug development.

Keyphrases

clinical trial
end stage renal disease
chronic kidney disease
newly diagnosed
peritoneal dialysis
electronic health record
emergency department
big data
deep learning
phase ii
patient reported