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Authors
Pini, Veronica
Catapano, Francesco
Bonaccorso, Rosa
Weisburd, Ben
Previtali, Stefano C.
Muntoni, Francesco
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Funding
Funding for this research was provided by:
CureCMD
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Article History
Received: 26 September 2025
Accepted: 9 January 2026
First Online: 3 February 2026
Declarations
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: The use of human biological samples was ethically approved and consent for research was obtained to facilitate pharmacological, gene and cell therapy trials in neuromuscular disorders (NRES Committee London- West London & GTAC, REC reference number 06/Q0406/33) and to allow the use of muscle biopsy to study pathogenesis and the expression and regulation of genes associated with neuromuscular disorders (NRES Committee London—REC reference 13/LO/1894), in compliance with national guidelines regarding the use of human-derived biological samples for research. Written informed consent was obtained from patient’s guardians.
: Not applicable.
: The authors declare no competing interests.
