Received: 1 October 2019
Accepted: 5 February 2020
First Online: 27 February 2020
Compliance with ethical standards
: Dr. Landfeldt is an employee of ICON plc (Stockholm, Sweden), outside the submitted work. Professor Sejersen reports receipt of grants/research supports from Sanofi-Genzyme and PTC, and receipt of honoraria or consultation fees from Sanofi-Genzyme, PTC, Biogen, Roche, and Sarepta, outside the submitted work. Professor Kirschner reports grants, personal fees and non-financial support from Roche, personal fees from Avexis, grants, personal fees and non-financial support from Biogen, grants from Santhera, grants and personal fees from PTC, outside the submitted work. Dr. Thompson and Dr. McMillan report no disclosures. Professor Lochmüller is the Editor-in-chief for the Journal of Neuromuscular Diseases (IOS Press) and reports consultancy and financial support for research projects and clinical trials by AMO Pharma, Biogen, Desitin, GW Pharma, Pfizer, PTC Therapeutics, Roche, Santhera, Sarepta, Satellos, and Ultragenyx, outside the submitted work.