Claeys, Kristl G.
Kushlaf, Hani
Raza, Syed
Hummel, Noemi
Shohet, Simon
Keyzor, Ian
Kopiec, Agnieszka
Graham, Ryan
Fox, Brian
Schoser, Benedikt http://orcid.org/0000-0002-2757-8131
Funding for this research was provided by:
Amicus Therapeutics
Universitätsklinik München
Article History
Received: 5 October 2023
Accepted: 28 March 2024
First Online: 11 April 2024
Declarations
:
: This study used data from the PROPEL phase 3 study. PROPEL was designed and monitored in accordance with the ethical principles of Good Clinical Practice guidelines and the Declaration of Helsinki. The clinical study protocol was reviewed and approved by the appropriate independent ethics committee or institutional review board at each study site.
: All participants provided written informed consent before participating in the study.
: SR, SS, IK, BF, and RG are former or current employees of, and hold stock in, Amicus Therapeutics. NH and AK are employees of Certara; Certara is a paid consultant to Amicus Therapeutics. SR is now employee of Argenx. KGC received speaker’s honoraria and travel reimbursement from Amicus Therapeutics unrelated to this publication and is Chairholder of the Emil von Behring Chair for Neuromuscular and Neurodegenerative Disorders by CSL Behring unrelated to this publication. KGC and BS are members of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO-NMD). HK served as a consultant on advisory boards for Alexion Astrazeneca Rare Disease, Argenx, UCB, Immunovant, and Sanofi. BS serves as a consultant and advisor for Amicus, Argenx, Astellas, Bayer, Maze, Sanofi, Pepgen, and Taysha; he received unrestricted grant/Research Support from Amicus and Astellas.