An international consortium of researchers and clinicians involved in therapeutic trials in Duchenne muscular dystrophy (DMD) is calling for a methodological revision based on the following findings:
- Current clinical trials in DMD have so far been based on the constitution of patient groups that are as homogeneous as possible on the genotypic level.
- This is particularly true for “exon skipping” approaches.
- With the increase in the number of trials, it is becoming increasingly difficult to create control groups.
- this genetic component would finally only marginally intervene in the variations observed at the phenotypic level, at least over a twelve-month observation period.
The authors therefore suggest that this constraint should be overcome in the design of future comparative studies.
