Murrell, Dedee F.
Paller, Amy S.
Bodemer, Christine
Browning, John
Nikolic, Milos
Barth, Jay A.
Lagast, Hjalmar
Krusinska, Eva
Reha, Allen
,
Funding for this research was provided by:
Amicus Therapeutics, Inc. (Not applicable)
Article History
Received: 20 February 2020
Accepted: 5 June 2020
First Online: 21 July 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.
: DFM has served as a consultant and investigator for Amicus, Amryt, Castle Creek Pharma, and Shire and has conducted her own investigator-initiated randomized controlled trials in recessive dystrophic epidermolysis bullosa and epidermolysis bullosa simplex. 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. CB has served on advisory boards for Pfizer and 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. 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. JAB, HL, and AR are employees of and hold stock in Amicus. EK is a paid consultant to Amicus.