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First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD.

Laurent ServaisDamien EggenspielerMargaux PoleurMarc GreletFrancesco MuntoniPaul StrijbosMélanie Annoussamy
Published in: Nature medicine (2023)
Keyphrases
  • duchenne muscular dystrophy
  • transcription factor
  • combination therapy
  • muscular dystrophy
  • replacement therapy