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Article History

Received: 18 December 2019

Revised: 10 February 2020

Accepted: 11 February 2020

First Online: 21 March 2020

Compliance with ethical standards

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: Jean-Yves Hogrel is the inventor of the MyoGrip device (patented). Erik Niks reports consultancies for BioMarin, Summit and WAVE for which reimbursements were received by the LUMC. Imelda de Groot has received consulting and education fees from PTC Therapeutics, Santhera, Biomarin/Prosensa. Volker Straub received speaker honoraria from Sanofi Genzyme and has participated in advisory boards for Audentes Therapeutics, Biogen, Bristol-Myer Squibb, Exonics Therapeutics, Italfarmaco S.p.A., Pfizer, Roche, Sanofi Genzyme, Sarepta Therapeutics, Summit Therapeutics, UCB, and Wave Therapeutics. He has research collaborations with Ultragenyx and Sanofi Genzyme. Francesco Muntoni has received consulting fees from Biogen, Italfarmaco, Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, and Wave Therapeutics; and is involved as Chief Investigator in the EUFP7 / Sarepta Therapeutics funded clinical trial (SKIP NMD study, EU FP 7 No. HEALTH-F4-2012-30537) and principal investigator in the Sarepta Therapeutics funded study ESSENCE. He is also 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. The support of the MRC Neuromuscular Centre Biobank and of the Muscular Dystrophy UK is also gratefully acknowledged. Thomas Voit serves on scientific advisory boards for Constant Pharmaceuticals and Metriopharm; he is a co-founder of AlterGene; he serves or served as a consultant for Biomarin, Biophytis, Evox, Capricor, Catabasis, Debiopharm, Italfarmaco, Santhera, Sarepta, Servier, Solid and Summit. Teresa Gidaro has served on scientific advisory boards for PTC and Ultragenyx. Laurent Servais has received consulting fees from Roche, Biogen, Avexis, Cytokinetics, Sarepta, Biomarin, Santhera, Servier, Biophytis and Dynacure. He is coordinating natural history studies funded by Valerion, Dynacure and Roche. The other authors report no disclosure.

: All studies have been approved by the appropriate ethics committee and have, therefore, been performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments. All patients or legal guardians for patients younger than 18 years of age gave written informed consent prior to participation in the studies.