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Article History

Received: 27 March 2025

Accepted: 9 October 2025

First Online: 16 December 2025

Declarations

:

: All investigations were performed according to the Declaration of Helsinki. All investigations were performed after approval by the respective ethics committees of the participating centers (project number: 2016-401-f-S, ).

: Informed consent was obtained from all subjects involved in the study.

: ML received speaker honoraria, travel funding, and research grants from Amicus Therapeutics, Sanofi, Chiesi, and Takeda. SCK received speaker honoraria, travel funding from Amicus Therapeutics, Sanofi, Chiesi, and Takeda. CK received speaker honoraria and travel support from Amicus Therapeutics, Chiesi, Sanofi, and Takeda. NM is a consultant for and/or received speak-er’s honoraria and travel grants from Amicus Therapeutics, Biomarin, Chiesi, JCR, Sanofi, Takeda, and Orphazyme. JBH received consulting fees and/or honoraria from Chiesi, Sanofi, Takeda, Amicus, and Immedica and travel support from Chiesi and Amicus. MCy received speaker honoraria/travel grants from Takeda, Amicus Therapeutics, Idorsia, and Alexion. JK received speaker honoraria and/or travel funding from Amicus Therapeutics, Sanofi, Chiesi, Novartis, Takeda, and Alexion (Astrazeneca Rare Disease). EB received research grants and speaker honoraria from Sanofi, Takeda, Chiesi and Amicus Therapeutics. AB received speaker honoraria from Amicus Therapeutics and Biogen. MC, PL, and MM report no conflicts of interest.