Antisense and gene therapy trials in Duchenne muscular dystrophy

Mariacristina Scoto1, Francesco Muntoni1

  • 1Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health and Institute of Neurology, University College London, London, UK.

Med (New York, N.Y.)
|December 13, 2025
PubMed