Funding for this research was provided by:
Amicus Therapeutics, Inc. (Not applicable)
Received: 20 February 2020
Accepted: 25 May 2020
First Online: 23 June 2020
Ethics approval and consent to participate
: The study was conducted in accordance with ethical principles founded in the Declaration of Helsinki and in compliance with the protocol, International Conference on Harmonisation Good Clinical Practice, and applicable local legal and regulatory requirements. An Institutional Review Board/Independent Ethics Committee reviewed and approved the study design and protocol at each study site, and signed informed consent/assent was obtained from each patient (or legal guardian if under 18 years of age) prior to entering the study.
: Not applicable.
: ASP has served as an investigator (uncompensated) for Amicus, Castle Creek Pharmaceuticals, Lenus Therapeutics, and Shire and as a consultant with honorarium for Castle Creek Pharmaceuticals, Lenus Therapeutics, and MEDACorp. JB has served as an investigator for Amicus, Galderma, Valeant, Patagonia, and Regeneron and as a speaker for Medimetriks and Promius. MN has received honoraria from Meda/Mylan, Medis, La Roche-Posay/L’Oreal, Uriage, Takeda, Novartis, Janssen, and Amicus. CB has served on advisory boards for Sanofi, as a consultant for Expanscience, as an investigator (uncompensated) for Amicus, as a speaker for La Roche-Posay and Expanscience, and has received honoraria from Celgene and Novartis. DFM has served as a consultant and investigator for Amicus, Amryt, Castle Creek Pharmaceuticals, and Shire and has conducted her own investigator-initiated randomized controlled trials in recessive dystrophic epidermolysis bullosa and epidermolysis bullosa simplex. EK is a paid consultant of Amicus. AR, HL, and JAB are employees of and hold stock in Amicus.