Faced with the difficulties and sometimes disappointed expectations of certain therapeutic trials in Duchenne muscular dystrophy (DMD), an international consortium of researchers sought to refine the individual trajectories of the patients concerned:
- the study involved 51 DMD patients (aged between 7 and 18 years) and focused on the period from the loss of walking ability,
- various parameters of strength and function (including lung function) were analysed before and after loss of walking,
- some of these parameters were found to be predictive, such as the decrease in respiratory vital capacity and the upper limb functional score (ULS),
The authors suggest including patients with such trajectories in the group of non-ambulatory patients used for the trials.
