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Approval was granted by the Ethics Committee of Coordinating Center (Gemelli Hospital, n\u00b0 0033773\/22-Oct 26 th 2022).","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"Written informed consent was obtained from all individual participants\/their parents or caregiver included in the study.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}},{"value":"Coratti G, D\u2019Amico 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.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"410"}}