The ability of a composite prognostic score to predict time to loss of ambulation (LoA) in patients with Duchenne muscular dystrophy (DMD) has been validated in an independent dataset, researchers report.
The benefits of individualized prediction of time to LoA include improved counselling for patients and caregivers, who may need to order a power wheelchair or modify their home for accessibility. Better prediction could also improve patient selection for clinical trials and help measure long-term outcomes.