Doorenweerd, Nathalie http://orcid.org/0000-0003-2932-4327
de Rover, Mischa http://orcid.org/0000-0001-5703-666X
Marini-Bettolo, Chiara
Hollingsworth, Kieren G. http://orcid.org/0000-0002-3135-8131
Niks, Erik H. http://orcid.org/0000-0001-5892-5143
Hendriksen, Jos G. M.
Kan, Hermien E. http://orcid.org/0000-0002-5772-7177
Straub, Volker http://orcid.org/0000-0001-9046-3540
Funding for this research was provided by:
Duchenne Parent Project
Gratama Stichting (10.13)
Muscular Dystrophy UK (RA3/3079/1)
Article History
Received: 5 December 2019
Revised: 28 July 2020
Accepted: 24 November 2020
First Online: 3 January 2021
Compliance with ethical standards
:
: This study was supported by Duchenne Parent Project Netherlands (grant Brain imaging and cognition in Duchenne muscular dystrophy – 2010); Muscular Dystrophy UK (grant no. RA3/3079/1); and Gratama Foundation Netherlands (Leiden University, grant no. 10.13). The sponsors had no role in study design, data collection, data analyses, data interpretation, or writing of the report. The corresponding author had full access to all the data in the study and had final responsibility for the decision to submit for publication. This study took place in two sites, at LUMC, Leiden, The Netherlands and Newcastle University, Newcastle upon Tyne, in the United Kingdom, and was approved by the local medical ethic committees in both sites. Participants were recruited from patient registries, local clinics and flyers. Written informed consent was received from all participants and/or their legal guardians. ND reports grants from Duchenne Parent Project and grants from Gratama Stichting during the conduct of the study; EN report grants from Duchenne Parent Project, ZonMW and AFM, consultancies for BioMarin and Summit, and worked as local investigator of clinical trials of BioMarin, GSK, Lilly, Santhera, Givinostat, and Rocheoutside the submitted work. KGH reports grants from the United Kingdom Medical Research Council, Diabetes UK, the European Union (H2020, 667,078) and the Newcastle Healthcare Charity, consultancy for Summit pharmaceuticals and trial support from ImagingDMD-UF outside the submitted work. HK reports grants from ZonMW, AFM, Duchenne Parent Project, Gratama Stichting, NWO and the EU and research support from Philips Healthcare during the conduct of the study, consultancy for BioMarin and aTyr Pharma, and trial support from ImagingDMD-UF outside the submitted work. All reimbursements were received by the LUMC or Newcastle University. No personal financial benefits were received. VS has participated in advisory boards for Audentes Therapeutics, Biogen, Exonics Therapeutics, Italfarmaco S.p.A., Roche, Sanofi Genzyme, Sarepta Therapeutics, Summit Therapeutics, UCB, and Wave Therapeutics. He has research collaborations with Ultragenyx and Sanofi Genzyme. Several of the authors are members of the European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD]. All other authors have nothing to declare.