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Article History

Received: 3 March 2025

Accepted: 24 April 2025

First Online: 5 June 2025

Declarations

:

: Inês Rua has received honoraria for lectures and/or travel support from BioMarin and Ascendis; and has been an investigator in observational studies and/or clinical trials for Pfizer, Ascendis, BioMarin, and QED. Isabel Silva has received travel support from BioMarin; and has been an investigator in observational studies for BioMarin. Upon completion of the manuscript, Isabel Silva has changed affiliation to Unidade Local de Saúde de São José, Lisbon, Portugal. Christoph Beger has received support for attending meetings and/or travel from BioMarin. Cristina Gomes has received honoraria for lectures and/or travel support from BioMarin; and has been research nurse in observational studies or clinical trials for Pfizer, Ascendis, BioMarin, and QED. Maria J. Pais has been research nurse in observational studies and/or clinical trials for Pfizer, BioMarin, and QED. Alice Mirante has received honoraria for lectures, advisory boards, and/or travel support from BioMarin; and has been an investigator in observational studies and/or clinical trials for Pfizer, Ascendis, and BioMarin. Sérgio B. Sousa has received honoraria for lectures, advisory boards, and/or travel support from BioMarin, Ascendis, and Kiowa Kirin; and has been an investigator in observational studies and/or clinical trials for Pfizer, Ascendis, BioMarin, and QED.

: The study was conducted in accordance with the Declaration of Helsinki, and approval from Unidade Local de Saúde de Coimbra ethics committee was obtained (ref. OBS.SF.140-2022; no. 490/CE; 1 August, 2024). Written consent was obtained for the use of any identifying information.