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Article History

Received: 6 December 2023

Accepted: 18 February 2025

First Online: 11 March 2025

Declarations

:

: Not applicable.

: Not applicable.

: IA, TBO, SB, VCD, BF, SF, PK, and GM have no conflict of interest to declare. EGN has received payment or honoraria for lectures/presentations, support for attending meetings and/or travel, and has participated in on a Data Safety Monitoring Board or Advisory Board for BioMarin. MAS has received payment or honoraria for lectures/presentations, support for attending meetings and/or travel from BioMarin and Sanofi, not related to this work. MI has received payment or honoraria for lectures/presentations from BioMarin. CL has received payment or honoraria for lectures/presentations. EL has received grants or contracts from EC and DFG. He has also received consulting fees, payment or honoraria for lectures/presentations, support for attending meetings and/or travel and has participated in a Data Safety Monitoring Board or Advisory Board for BioMarin. MM has received grants or contracts paid to his institution from from Merck Serono, Pfizer, and Novo Nordisk. He has received consulting fees from Novo Nordisk, Pfizer, and BioMarin, as well as payment or honoraria for lectures/presentations from Novo Nordisk, Sandoz, Merck Serono, Pfizer, Ascendis, and BioMarin. KM has received grants or contracts, payment or honoraria for lectures/presentations, support for attending meetings and/or travel and has participated in a Data Safety Monitoring Board or Advisory Board for BioMarin. ZP has received consulting fees and payment or honoraria from Newclip Technics France. MS has received grants or contracts and payment for expert testimony from BioMarin paid to Association AISAC ODV. SS has received grants/contracts from Ascendis and Pfizer. He has also received payment or honoraria for lectures/presentations, support for attending meetings and/or travel, and participated in a Data Safety Monitoring Board or Advisory Board for BioMarin.