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Authors
Baujat, Genevieve
Hamandjian, Marc-Antoine
Jannot, Anne-Sophie
Karam, Pierre
Cormier-Daire, Valérie
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Funding
Funding for this research was provided by:
BioMarin Pharmaceutical (BioMarin Pharmaceutical)
French Ministry of Health (French Ministry of Health)
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Article History
Received: 23 June 2025
Accepted: 25 September 2025
First Online: 3 November 2025
Declarations
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: This non-interventional study was approved by the BNDMR scientific committee (BPD-2018-DIAG-008/-002) and registered with the French Data Protection Authority (CNIL: MR004) and the AP-HP local registry (#20230314171333). In accordance with French law, patients were informed about the inclusion of their data in BNDMR and had the opportunity to object to its use for research. Only those who did not object were included in our study.
: All authors agree with the content of the manuscript. All authors have agreed to the submission and publication of this manuscript.
: GB declares competing interests for advisory consultancy and expert report production for BioMarin, Inozyme, Ipsen, Sanofi, and Alexion; participation in scientific work for Incyte, Ipsen, QED, Ascendis, BioMarin, Alexion, Clementia, and Therachon Pfizer; and writing articles and interventions for Elsevier, BioMarin, Ipsen, and Alexion. MAH is a BioMarin employee and has stock options. ASJ and PK have no conflicts of interest to declare. VCD declares competing interests related to advisory consultancy or interventions for BioMarin and QED.
