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Authors
Allegri, Anna Elsa Maria
Bedeschi, Maria Francesca https://orcid.org/0000-0002-6723-9177
Bocchi, Maria Beatrice
Camurri, Valentina
Gonfiantini, Michaela Veronika
Leoni, Chiara
Mariani, Milena
Palmacci, Osvaldo
Porro, Matteo
Riganti, Simone
Tedesco, Caterina
Rinaldi, Berardo
Scarano, Emanuela
Schiavariello, Concetta
Selicorni, Angelo
Stagi, Stefano
Verdoni, Fabio
Zampino, Giuseppe
Maghnie, Mohamad
Onesimo, Roberta
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Funding
Funding for this research was provided by:
ENDO ERN and ERN BOND - European Reference Network for rare Bone Diseases (ENDO ERN and ERN BOND - European Reference Network for rare Bone Diseases)
BioMarin Pharmaceutical (None)
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Article History
Received: 28 June 2024
Accepted: 8 June 2025
First Online: 17 July 2025
Declarations
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: The local ethics committee approved the use of vosoritide. Owing to its retrospective nature, no ethics committee approval was necessary for this study.
: Informed consent for publication was obtained from all patients.
: AEM Allegri declares the following conflict of interest: lecture and advisory board fees: BioMarin; financial support for training conferences: BioMarin, Pfizer and Sandoz. MF Bedeschi has received funding as a principal investigator for Sanofi, Biomarin, and QED clinical trials and from Biomarin as speaker in sponsored symposia and from Biomarin as a member of the Advisory Board. M. V. Gonfiantini has received funding as a principal investigator for Amgen and Biomarin clinical trials and for participating in advisory boards on Achondroplasia for Biomarin. M Maghnie received Grant support from Pfizer, Novo Nordisk and Merck Serono and consultancy. honoraria and speaker fees from Pfizer, Merck Serono, Novo Nordisk, Sandoz, and BioMarin. M Mariani declares the following conflicts of interest: Biomarin, Pfizer. R Onesimo received funding as the principal investigator for Sanofi, Biomarin, and QED clinical trials from Biomarin as speaker in sponsored symposia and from Biomarin as a member of the advisory board. B Rinaldi declares the following conflicts of interest: Biomarin, Sanofi, and QED clinical trials. A Selicorni declares the following conflicts of interest: Biomarin, Pfizer. G Zampino has received funding as a principal investigator for Pfizer clinical trials. MB Bocchi, V. Camurri, C. Leoni, O. Palmacci, M. Porro, S. Riganti, E. Scarano, and C. Schiavarello have no conflicts of interest to declare.
