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Article History

Received: 30 October 2024

Accepted: 6 April 2025

First Online: 15 May 2025

Declarations

:

: Not applicable.

: Not applicable.

: BF: None to declare. MA: None to declare. TBO: None to declare. SB: Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from BioMarin. Support for attending meetings and/or travel from BioMarin. Participation on a data safety monitoring board or advisory board from BioMarin. MB: All support for the present manuscript from CESAS Medical. VCD: Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from BioMarin. Participation on a data safety monitoring board or advisory board from BioMarin. SF: None to declare. EGN: Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from BioMarin. Support for attending meetings and/or travel from BioMarin. MI: Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from the EAF. Support for attending meetings and/or travel from the EAF. PK: Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from BioMarin. NM: None to declare. MM: Grant support from Pfizer, Novo Nordisk, and Merck Serono. Consulting fees and payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from Pfizer, Novo Nordisk, Sandoz, Merck Serono, and BioMarin. Participation on a data safety monitoring board or advisory board from Pfizer. JM: All support for the present manuscript from CESAS Medical. Support for attending meetings and/or travel from CESAS Medical. KM: All support for the present manuscript from BioMarin. Grants or contracts from any entity from BioMarin. Consulting fees from QEDBiobridge. Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from BioMarin and Novo Nordisk. Participation on a data safety monitoring board or advisory board from QEDBiobridge. Leadership or fiduciary role in other board, society, committee, or advocacy group, paid or unpaid from BKMF e.V. GM: None to declare. LN: Consulting fees from Fidia Pharma, and Procter & Gamble. Receipt of equipment, materials, drugs, medical writing, gifts, or other services from Jazz Pharmaceuticals. ZP: Consulting fees from Orthofix Italy. Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from Orthofix Italy and Newclip France. Support for attending meetings and/or travel from Orthofix Italy. MS: Grants or contracts from any entity from BioMarin and QEDBiobridge to AISAC. Payment for expert testimony from BioMarin and QEDBiobridge to Marco Sessa. Support for attending meetings and/or travel from BioMarin and QEDBiobridge to Marco Sessa and AISAC. Participation on a data safety monitoring board or advisory board from BioMarin to AISAC. SBS: Payment or honoraria for lectures, presentations, speaker’s bureaus, manuscript writing or educational events from BioMarin. Support for attending meetings and/or travel from BioMarin. Participation on a data safety monitoring board or advisory board from BioMarin