Real-world study predicts clinically significant milestones in patients with DMD

Researchers from multiple centers in London and Paris have reported sensitive motor function outcomes in non-ambulatory Duchenne muscular dystrophy (DMD) patients with high standardized response mean (SRM) using a combination of tools measuring muscle strength and structure.

Presenting at the WMS 2021 virtual congress, Ms Charlotte Lilien from the MDUK Oxford Neuromuscular Centre, University of Oxford, UK, noted that there was a lack of longitudinal, prospective natural history studies on upper limb disease evolution in non-ambulatory DMD patients.