Bruckner, Anna L.
Losow, Michael
Wisk, Jayson
Patel, Nita
Reha, Allen
Lagast, Hjalmar
Gault, Jamie
Gershkowitz, Jayne
Kopelan, Brett
Hund, Michael
Murrell, Dedee F.
Funding for this research was provided by:
Amicus Therapeutics, Inc. (NA)
Article History
Received: 16 July 2019
Accepted: 16 December 2019
First Online: 3 January 2020
Ethics approval and consent to participate
: The survey was approved by a central institutional review board (New England Independent Review Board). All patients or caregivers provided informed consent to participate in the survey.
: Not applicable.
: ALB has served as a consultant and an investigator for Amicus Therapeutics/Scioderm and Castle Creek, and as an investigator for Amryt, Fibrocell, ProQR, and Phoenix Tissue Repair.JW, NP, AR, HL, ML, and JGa, are employees of and hold stock in Amicus Therapeutics.JGe is the co-founder and vice chair on the board of Profession Patient Advocates in Life Sciences; a member of the board of trustees for the Healthcare Institute of New Jersey; a member of the board of directors for the Together Strong Foundation for NPC, and an employee of, and a stock holder and patent holder in, Amicus Therapeutics.BK is an employee of Debra of America.MH is an employee of EB Research Partnership.DFM has served as an investigator for Amicus Therapeutics, Amryt, Castle Creek, and Shire, and has served as an advisor for Amicus and Shire.