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Authors
Degan, Chiara https://orcid.org/0009-0001-9993-9346
Tobin, Rebecca A.
de Vries, Sharon I.
Jiménez-Requena, Albert
Peco, Amela
Guglieri, Michela
Diaz-Manera, Jordi
van der Burgt, Yuri E. M.
van Vlijmen, Bart J. M.
,
Bushby, Kate
McDermott, Michael P.
Hart, Kimberly A.
Tawil, Rabi
Martens, William B.
Herr, Barbara E.
McColl, Elaine
Speed, Chris
Wilkinson, Jennifer
Kirschner, Janbernd
King, Wendy M.
Eagle, Michelle
Brown, Mary W.
Willis, Tracey
Griggs, Robert C.
Straub, Volker
van Ruiten, Henriette
Childs, Anne-Marie
Ciafaloni, Emma
Shieh, Perry B.
Spinty, Stefan
Maggi, Lorenzo
Baranello, Giovanni
Butterfield, Russell J.
Horrocks, I. A.
Roper, Helen
Alhaswani, Zoya
Flanigan, Kevin M.
Kuntz, Nancy L.
Manzur, Adnan
Darras, Basil T.
Kang, Peter B.
Morrison, Leslie
Krzesniak-Swinarska, Monika
Mah, Jean K.
Mongini, Tiziana E.
Ricci, Federica
von der Hagen, Maja
Finkel, Richard S.
O’Reardon, Kathleen
Wicklund, Matthew
Kumar, Ashutosh
McDonald, Craig M.
Han, Jay J.
Joyce, Nanette
Henricson, Erik K.
Schara-Schmidt, Ulrike
Gangfus, Andrea
Wilichowski, Ekkehard
Barohn, Richard J.
Statland, Jeffrey M.
Campbell, Craig
Vita, Giuseppe
Vita, Gian Luca
FHoward, James Jr
Hughes, Imelda
McMillan, Hugh J.
Pegoraro, Elena
Bello, Luca
Burnette, WBryan
Thangarajh, Mathula
Chang, Taeun
Hathout, Yetrib
Szigyarto, Cristina Al-Khalili
Dang, Utkarsh J.
Tsonaka, Roula
Spitali, Pietro
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Funding
Funding for this research was provided by:
National Institutes of Health (R61NS119639)
Parent Project Muscular Dystrophy (22.010)
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Article History
Received: 11 February 2026
Accepted: 29 June 2026
First Online: 13 July 2026
Declarations
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: The FOR-DMD study was registered at ClinicalTrials.gov (no. NCT01603407). Serum samples and clinical data were collected following approval from the competent ethics committee at each institution in accordance with the International Conference on Harmonisation guidelines for Good Clinical Practice and the World Medical Association Declaration of Helsinki. Central Institutional Review Board (IRB) approval for the original FOR-DMD study was obtained from the University of Rochester Ethics Committee/IRB and Newcastle Clinical Trial Unit (NCTU) before distribution of study documents to each investigative site and enrollment of participants. Informed written consent for participation in biomarker research was obtained from the parents or legal guardians of participants at the time of enrollment in the clinical study. For the data collected retrospectively at Leiden University Medical Center (LUMC), written informed consent was obtained from all participants or their legal guardians in accordance with protocol B22.013, approved by the regulatory board at LUMC, called "Medische Ethische Toetsings Commissie" (METC, medical ethics review committee).
: Not applicable.
: The authors declare no competing interests.
