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Article History

Received: 21 July 2025

Accepted: 8 February 2026

First Online: 1 April 2026

Declarations

:

: This non-interventional study was conducted in accordance with the principles laid down by the 18th World Medical Assembly (Helsinki, 1964) and all applicable amendments laid down by the World Medical Assemblies, the International Conference for Harmonisation Good Clinical Practice guidelines, and in compliance with French laws and regulations. As required by local regulations, the protocol for the study and subsequent amendments (in this case to prolong recruitment, add/remove sites, or change investigators) were submitted to the appropriate ethics committee for approval (Comité de Protection des Personnes Sud Méditerranée IV, Montpellier, France; initial approval date 9 October 2018; CPP reference 18 09 03). All participants were informed to the fullest extent possible about the details of the study, and a written non-opposition form was signed by the participant or legally acceptable representative prior to participation in the study; for minors, non-opposition of at least one parent or holder of parental authority was required.

: Not applicable for the aggregate data reported here. All individuals named in the Acknowledgements section have provided written consent.

: OL: travel grants and speaker honoraria from Amicus, Chiesi, and Genzyme/Sanofi. AM: research grants from Amicus. GP: travel grants, advisory board honoraria and speaker honoraria from Amicus. DL: participation in advisory board and congress activities organized by Amicus, Chiesi, Sanofi, and Takeda. BD: fees for advisory board participation from Amicus. SB: research grants and speaker honoraria from Amicus, Chiesi, Sanofi, and Takeda. AH: consulting/advisor fees from Alnylam, Amicus Therapeutics, Bayer, Cytokinetics, MyoKardia/Bristol Myers Squibb, Pfizer, Sanofi Genzyme, and Tenaya Therapeutics. CM: employee and shareholder of Amicus Therapeutics SAS. YF: employee of Soladis Clinical Studies, the contract research organization in charge of managing the study. AF: research grants and travel grants from Amicus and Sanofi. EN: advisory boards and congress invitations from Amicus and Sanofi.