Schiffmann, Raphael
Bichet, Daniel G.
Jovanovic, Ana
Hughes, Derralynn A.
Giugliani, Roberto
Feldt-Rasmussen, Ulla
Shankar, Suma P.
Barisoni, Laura
Colvin, Robert B.
Jennette, J. Charles
Holdbrook, Fred
Mulberg, Andrew
Castelli, Jeffrey P.
Skuban, Nina
Barth, Jay A.
Nicholls, Kathleen
Funding for this research was provided by:
Amicus Therapeutics, Inc
Article History
Received: 29 November 2017
Accepted: 18 April 2018
First Online: 27 April 2018
Ethics approval and consent to participate
: Written consent to participate in the study was obtained from all patients. The study was approved by the institutional review board or ethics committee at each participating center and was conducted in accordance with the International Conference on Harmonisation Good Clinical Practice guidelines and the principles of the Declaration of Helsinki.
: RS has served as a consultant for and received research funding from Protalix Biotherapeutics and Amicus. DGB has received research funding, serves as a consultant, and is on the speaker’s bureau for Amicus and Genzyme, and has received research funding from Shire. AJ has received advisory honoraria and speaker’s fees from Shire, Amicus, Biomarin, and Genzyme. DAH has served as a consultant for and received research funding and honoraria from Amicus, Shire, Genzyme, Protalix, and Actelion. RG has received honoraria from Amicus, Biomarin, Genzyme, and Shire. UFR reports other support from Amicus during the conduct of the study, grant support and speaker’s honoraria from Amicus, Genzyme, and Shire HGT outside the submitted work, and research funding from Novo Nordisk Research Foundation. SPS reports grants and non-financial support from Amicus during the conduct of the study. LB has served as a consultant for Protalix. RBC has served as a consultant for Amicus and has received grants from the National Institutes of Health. JCJ does not have anything to disclose. KN has served as an advisor for Amicus, Shire HGT, and Genzyme, has received research support from Amicus and Shire HGT, and has received travel support from Genzyme. FH, AM, JPC, NS, and JAB are employees of and hold stock in Amicus.
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