Bravetti, Chiara
Coratti, Giorgia
Pera, Maria Carmela
Gadaleta, Giulio
Mongini, Tiziana
Coccia, Michela
Ferrero, Amanda
Costantini, Emanuele Maria
Longo, Antonella
Cumbo, Francesca
Catteruccia, Michela
D’Amico, Adele
Morando, Simone
Brolatti, Noemi
Bruno, Claudio
Verriello, Lorenzo
Pessa, Maria Elena
Antonaci, Laura
Faini, Claudia
Liguori, Rocco
Vacchiano, Veria
Ruggiero, Lucia
Zoppi, Dario
Russo, Anna
Torri, Francesca
Ricci, Giulia
Chiappini, Roberto
Siciliano, Gabriele
Trabacca, Antonio
Agosto, Caterina
Benedetti, Francesca
Pane, Marika
Mercuri, Eugenio
,
Capasso, Anna
Arpaia, Chiara
Cicala, Gianpaolo
Paolucci, Sophia
Cutrì, Chiara
Villa, Marianna
Sestito, Simona
Polo, Agnese
Falco, Anna
Rausa, Azzurra
Moro, Marianna
Porto, Chiara
De Gioia, Alessia
De Rosa, Elena
Rolleri, Elisa
Ascione, Federica
Giammartino, Federica
Petracca, Giorgia
Esposito, Lucia
Bianchetti, Maddalena
Marsella, Giuseppe
Malaspina, Martina
Dainesi, Irene
Mannucci, Sara
De Maria, Martina
De Luca, Giacomo
Mizzoni, Irene
Carlesi, Adelina
Funding for this research was provided by:
Roche Italia (SMA-NH-LT)
Article History
Received: 3 March 2025
Revised: 7 May 2025
Accepted: 16 May 2025
First Online: 10 June 2025
Declarations
:
: This study was performed in line with the principles of the Declaration of Helsinki. Approval was granted by the Ethics Committee of Coordinating Center (Gemelli Hospital, n° 0033773/22-Oct 26 th 2022).
: Written informed consent was obtained from all individual participants/their parents or caregiver included in the study.
: Coratti G, D’Amico A, Bruno C, Trabacca A, Maggi L, Pera MC, Ricci F, Mongini T, Pane M, Mercuri E report personal fees from BIOGEN S.R.L., ROCHE, AVEXIS and NOVARTIS outside the submitted work; Pane M and Mercuri E report personal fees from PTC THERAPEUTICS and SAREPTA outside the submitted work; Coratti G reports personal fees from GENESIS PHARMA and Biologix outside the submitted work; Bruno I reports personal fees from Biogen and Astrazeneca outside the submitted work; Mercuri E reports from personal fees SANTHERA outside the submitted work; Siciliano G and Ricci G received consulting fees from Biogen and Roche. Bravetti C, Gadaleta G, Coccia M, Ferrero A, Costantini E, Longo A, Catteruccia M, Morando S, Brolatti N, Verriello L, Cumbo F, Pessa ME, Antonaci L, Vacchiano V, Faini C, Liguori R, Ruggiero L, Zoppi D, Caterina Agosto, Francesca Benedetti, Russo A, Torri F, have nothing to disclose. Author Contributions: All authors contributed to the study conception and design. Material preparation, data collection and analysis were performed by Chiara Bravetti, Giorgia Coratti and Maria Carmela Pera. The first draft of the manuscript was written by Chiara Bravetti, Giorgia Coratti, Maria Carmela Pera, Eugenio Mercuri and all authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.