Digital outcome measures in Duchenne muscular dystrophy: Lessons learnt from clinical trials

Duchenne muscular dystrophy is a severe neuromuscular disorder characterized by progressive muscle degeneration resulting from mutations in the dystrophin gene. Digital outcome measures offer a promising alternative to traditional outcome measures used in clinical trials. This review explores the development and application of digital outcome measures in Duchenne muscular dystrophy, emphasizing the feasibility, reliability, sensitivity, and validity of these measures. The stride velocity 95th centile has been validated as a robust endpoint and has been approved for use in clinical evaluation of drugs for the treatment of Duchenne muscular dystrophy by the European Medicines Agency. Although digital outcome measures have the potential to enhance the efficiency and accuracy of clinical trials, challenges such as limited sample sizes and patient compliance persist. The integration of artificial intelligence into the data analysis is in progress, but further validation is required before these analysis strategies can be incorporated into future clinical trial methodologies.