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Authors
Schoser, Benedikt https://orcid.org/0000-0002-2757-8131
Attarian, Shahram
Graham, Ryan
Holdbrook, Fred
Goldman, Mitchell
Díaz-Manera, Jordi
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Funding
Funding for this research was provided by:
Amicus Therapeutics
Klinikum der Universität München
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Article History
Received: 25 July 2024
Revised: 14 October 2024
Accepted: 29 November 2024
First Online: 7 January 2025
Declarations
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: This study was supported by Amicus Therapeutics, Inc. Benedikt Schoser has received unrestricted research grants from Amicus Therapeutics, Inc., Astellas, Roche Diagnostics, Marigold Foundation, and AMDA Foundation and speaker’s honoraria from Amicus Therapeutics, Inc., Alexion, Kedrion, and Sanofi. He has participated as a scientific advisor for Amicus Therapeutics, Inc., Argenx, Astellas, Bayer, Maze, Pepgen, Sanofi, Spark, and Taysha. He declares no stocks or shares. Shahram Attarian has received consulting fees/honoraria from Sanofi, Amicus Therapeutics, Inc., Argenx, and Alexion and grant support from Biogen. He has received payment for speaking from Sanofi, Pfizer, LFB, Argenx, Alexion, Janssen, Alnylam, Novartis, Biogen, and Roche. Ryan Graham and Fred Holdbrook are employees of, and hold stock in, Amicus Therapeutics, Inc. Mitchell Goldman is a former employee of Amicus Therapeutics, Inc. Jordi Díaz-Manera has received consulting fees/honoraria from Amicus, Astellas, Sarepta, Sanofi, Argenx, and Lupin and grant support from Sanofi, Spark, Sarepta, and Boehringer Ingelheim. He has received payment for speaking from Sanofi, Sarepta, and Lupin.
: This study was conducted in accordance with ethical standards as laid down in the 1964 Declaration of Helsinki and its later amendments, and in compliance with the United States Food and Drug Administration regulations in 21 Code of Federal Regulations 56, European Union Clinical Trials Regulation 536/2014, and the International Council for Harmonisation Good Clinical Practice guidelines. For each study site, the clinical study protocol (and any amendments) and informed consent form were reviewed and approved by the appropriate independent ethics committee/institutional review board.
: All patients provided signed informed consent before any study-related procedures were performed. In Japan, the patient’s parental guardian (or legal representative) also had to sign the informed consent form for patients under 20 years of age.
