Towards a methodological flexibility of clinical trials in DMD?

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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.

 

DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials. Muntoni F, Signorovitch J, Sajeev G et al. Neurology. 2023 Feb.

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