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Article History

Received: 10 December 2024

Accepted: 19 March 2025

First Online: 6 May 2025

Declarations

:

: No individual persons provided data for this study. The research used publicly available data from published observational studies. Individual patient consent was not required for this secondary study using previously published observational studies.

: Not applicable. This manuscript contains no individual person’s data in any form.

: Roberto Giugliani has received unrestricted research grants from Azafaros, Chiesi, Cyclo, Instituto JCR, Jo Clemente Institute, Novartis, Paradigm, PassageBio, RegenxBio, Takeda and Sanofi, and speaker’s honoraria from Astellas, Chiesi, JCR, Pfizer, Takeda, Regenxbio, Sanofi and Ultragenyx. He has participated as a scientific advisor for Amicus, JCR, PTC, RegenxBio, Takeda and Ultragenyx He declares no stocks or shares. Hani Kushlaf served as a consultant on advisory boards for Alexion AstraZeneca Rare Disease, Amgen, Catalyst Pharmaceuticals, and UCB. Erica Wright has received a grant from the U.S. Health Resources and Services Administration, honoraria for teaching at Metabolic University, and support for meeting-related travel from the Association of Public Health. Edmar Zanoteli has received consulting fees and payment/honoraria from Sanofi, and participated on data safety monitoring boards for Sanofi. Faryn Solomon and Tmirah Haselkorn are employees of Astellas Pharma Global Development. Benedikt Schoser has received unrestricted research grants from Roche Diagnostics, Marigold Foundation Canada, AMDA Foundation USA, EU Horizon ComPASs and PaLaPin, and speaker’s honoraria from Amicus, Alexion, Kedrion, Sanofi. He has participated as a scientific advisor for Amicus, Alexion, Argenx, Astellas, Avidity, Pepgen, Sanofi. He declares no stocks or shares.