Harfouche, Melodi
Kishnani, Priya S.
Krusinska, Eva
Gault, Jamie
Sitaraman, Sheela
Sowinski, Amanda
Katz, Irina
Austin, Stephanie
Goldstein, Margi
Mulberg, Andrew E.
Funding for this research was provided by:
Amicus Therapeutics, Inc. (Not applicable)
Article History
Received: 5 May 2020
Accepted: 15 September 2020
First Online: 9 October 2020
Ethics approval and consent to participate
: The clinical study was approved by the Duke University Institutional Review Board. All participants provided informed consent to be included in the study.
: Not applicable.
: MH, JG, MG, AS, SS, and AEM are employees of and hold stock in Amicus. EK is a paid consultant for Amicus. IK is a BioNJ fellow partially sponsored by Amicus.PK received research grants from Sanofi Genzyme, Valerion, and Amicus and consulting fees and honoraria from Sanofi Genzyme, Amicus, Vertex, and AskBio; served on the advisory boards of Amicus, Baebies, and Sanofi Genzyme; and holds stock in AskBio. SA received honoraria and funding for travel from Sanofi Genzyme and funding for travel from Valerion.