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Article History

Received: 12 September 2025

Accepted: 23 February 2026

First Online: 9 March 2026

Declarations

:

: All investigations were performed after approval by the respective ethics committees of the participating centers (project number: 2011–347-f-s).

: Written informed consent for publication was obtained from patients where appropriate.

: Malte Lenders received speaker honoraria, travel funding, and research grants from Amicus Therapeutics, Sanofi, Chiesi, Sumitomo Pharma, and Takeda. Eva Brand received research grants and speaker honoraria from Sanofi, Takeda, Chiesi, Eleva, and Amicus Therapeutics. Albina Nowak received speaker honoraria and research grants from Amicus, Takeda, and Sanofi. Christine Kurschat received speaker honoraria and travel support from Amicus Therapeutics, Chiesi, Eleva, Sanofi, and Takeda. Markus Cybulla received speaker honoraria/travel grants from Takeda, Amicus Therapeutics, Idorsia, and Alexion. Sima Canaan-Kühl received speaker honoraria and travel funding from Sanofi, Takeda, Chiesi, Eleva, and Amicus Therapeutics. Fabian Braun received speaker honoraria, travel funding, and research grants from Amicus Therapeutics, Sanofi, Chiesi, and Takeda. Constantin Gatterer received speaker honoraria and/or research grants and/or travel grants from Amicus Therapeutics, Chiesi, Sanofi-Genzyme, and Takeda. Gere Sunder-Plassmann received honoraria, travel grants, study fees or research grants from Amicus Therapeutics, Sanofi-Genzyme, Chiesi, Freeline, and Idorsia. Victoria Sokalski received speaker honoraria, travel funding, and research grants from Amicus Therapeutics, Sanofi, Takeda, and Chiesi. Peter Nordbeck received speaker/advisory board honoraria and research grants from Amicus Therapeutics, Boehringer Ingelheim, Chiesi, Idorsia, Pfizer, Sangamo, Sanofi-Genzyme, and Takeda. Christoph Wanner received speaker/advisory board honoraria from Amicus Therapeutics, Boehringer Ingelheim, Chiesi, Idorsia, and Sanofi-Genzyme.