Given the relative failure of a certain number of therapeutic trials, an international consortium of specialists in Duchenne muscular dystrophy (DMD), in collaboration with the AFM-Téléthon in particular, has examined the motor trajectories in this pathology:
- to this end, they gathered data and clinical assessments from more than 1,000 DMD patients, in the context of trials or real-life follow-up,
- the subjects were aged between 4 and 18, still able to walk and receiving long-term corticosteroids,
- significant thresholds of change were defined with a confidence interval set at 80%,
- these were 2.8 points for the North Star scale, 1.3 seconds for the four-step ascent test and 36.3 metres for the six-minute walk test.
The authors consider that above these thresholds, the improvement observed is indeed due to the treatment.
