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Article History

Received: 7 February 2025

Revised: 25 April 2025

Accepted: 28 April 2025

First Online: 7 June 2025

Declarations

:

: Pietro Riguzzi has received funding from Edgewise therapeutics through Newcastle University. Meredith K. James provides consultancy services for the following companies: ATOM International. MKJ has participated on Advisory Boards for F. Hoffman La Roche AG, Sarepta Therapeutics and consultancies with Sarepta Therapeutics, Amicus, Edgewise, Genethon and Sanofi with fees paid to Newcastle University, and received fee support for PhD studies from the Jain Foundation. John Bourke serves on the Data Monitoring Committee for Sarepta Therapeutics DMD gene therapy trials, occasionally serves on the BMD trial design committee for Sarepta Therapeutics, and is an occasional external expert for AstraZeneca; JB also occasionally serves on the Pfizer stem cell therapy DMD trials advisory board, the EspeRare advisory panel, and the TREAT-NMD submission evaluation panel. Chiara Marini Bettolo has received a grant by Duchenne UK through Newcastle University. Robert Muni-Lofra has been participating in advisory boards for Biogen, Roche and Novartis and has delivered consultancy work for Pfizer, Italfarmaco, Sarepta, Summit and NS Pharma. Marianela Schiava has received a grant by UK Duchenne through Newcastle University. Luca Bello acknowledges research support from PTC Therapeutics; payments or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from PTC Therapeutics and Pfizer; payments for expert testimony from PTC Therapeutics; payments for participation on a Data Safety Monitoring Board or Advisory Board from PTC Therapeutics, Edgewise Therapeutics, and Roche. Elena Pegoraro acknowledges payments or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Biogen, Alexion and Roche; support for attending meetings and/or travels from Roche, Biogen, Argenx and Alexion; payments for participation on a Data Safety Monitoring Board or Advisory Board from Alexion, UCB Biopharma, Santhera and Sanofi. Michela Guglieri is a member of the advisory boards for Pfizer, NS Pharma, Dyne, and Roche (through Newcastle University); a member of the DSMB for Antisense Therapeutic (through Newcastle University); has received speaker honoraria (personal) from Sarepta, Italfarmaco, and Biogen; has had research collaborations (through Newcastle University) with PTC Therapeutics, ReveraGen, Roche, and Edgewise; and has been Principal Investigator for clinical trials sponsored by Pfizer, Edgewise, Dyne, Roche, Italfarmaco, and ReveraGen. The rest of the authors report no competing interests.

: The study was approved as a service review under the Newcastle upon Tyne Trust Hospital because the project was to evaluate the current natural history data to inform decision-making process on care management for patients with BMD.