Funding for this research was provided by:
Cancer Research UK (Cl 15121 A 20256)
Sarepta Therapeutics Inc
Received: 31 January 2020
Accepted: 16 March 2020
First Online: 17 April 2020
Ethics approval and consent to participate
: Skeletal muscle biopsies were obtained from the MRC Centre for Neuromuscular Diseases Biobank London (Ethics ref.: 06/Q0406/33).
: Parents or legal guardians of children gave written informed consent for muscle biopsies to be used for research purposes.
: FM has received consulting fees from Biogen, Italfarmaco, Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, and Wave Therapeutics; and is supported by the National Institute of Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust, and University College London. All other authors declare that they have no competing interests.