Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy

Conclusions The group concluded that they were interested in pursuing such a modeling approach based on consideration of muscle fat fraction, timed function tests, muscle strength, as well as optimized scales of function in ambulant and non-ambulant patients, with additional consideration of time to event analyses of specific disease milestones. The variables that are finally included in the model will be dictated by the data – both what data is in the integrated dataset and what early stage analyses of that data tells us about what is most relevant to the final model. The context of use of the model would be to forecast changes in clinically meaningful outcome measures, which would inform clinical trial protocol development with respect to inclusion criteria, endpoints to include, and size and length of trials. Data Availability Statement This report is the proceedings of a meeting, and no new data was reported. Competing Interest Statement The authors have declared that no competing interests exist. Corresponding Author Jane Larkindale: jlarkindale@c-path.org
Source: PLOS Currents Muscular Dystrophy - Category: Neurology Authors: Source Type: research