Text and Data Mining valid from 2019-09-25
Received: 19 July 2019
Revised: 9 September 2019
Accepted: 10 September 2019
First Online: 25 September 2019
Compliance with ethical standards
: The authors have non-competing interests with regard to the present work. The authors declare no financial support for the conduct of the study. Dr. Scheidegger reports other from Sanofy Genzyme, outside the submitted work; Dr. Bello reports personal fees from PTC Therapeutics, grants from Santhera Pharmaceuticals to his Institution, personal fees from Sarepta Therapeutics, personal fees from Reveragen Biopharma, outside the submitted work; Dr. Pegoraro reports non-financial support from Genzyme, grants and non-financial support from Santhera, personal fees from Sarepta, personal fees and non-financial support from PTC Therapeuticals, personal fees and non-financial support from Roche, outside the submitted work; Maggie C. Walter has served on advisory boards for Avexis, Biogen, Novartis, Roche, Santhera, Sarepta, PTC Therapeutics, Ultragenyx, Wave Sciences, received funding for Travel or Speaker Honoraria from Novartis, Biogen, Ultragenyx, Santhera, PTC Therapeutics, and worked as an ad-hoc consultant for AskBio, Audentes Therapeutics, Biogen Pharma GmbH, Fulcrum Therapeutics, GLG Consult, Guidepoint Global, Gruenenthal Pharma, Novartis, Pharnext, PTC Therapeutics, and Roche. The remaining authors have nothing to disclose.
: The study has been approved by ethics committees of all the involved institutions. All data were gathered after informed consent was obtained from each participant, in accordance with specific national laws and the ethics standards laid down in the 1964 Declaration of Helsinki and its later amendments.