Funding for this research was provided by:
Sarepta Therapeutics, Inc (HEALTH-F4-2012-30537)
Cancer Research UK (Cl 15121 A20256)
Received: 11 December 2020
Accepted: 13 December 2020
First Online: 6 January 2021
: 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. LS received consulting fees from Sarepta, Roche, Pfizer, Novartis, Cytokynetics, Biogen, Regenexbio, Santhera, Affinia, Dynacure and Audentes. The views expressed in this paper are those of the authors and not necessarily those of the NHS, the NIHR, or the Department of Health.
: The study was conducted in accordance with the principles of the Declaration of Helsinki. Ethical approval was obtained in each of the participating centres (Newcastle and London, UK; Paris, France; and Rome, Italy)
: Parents or legal guardians of all patients provided written informed consent before study participation and genetic testing with all patients providing written assent.