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Authors
Catapano, Francesco
Alkharji, Reem
Chambers, Darren
Singh, Simran
Aghaeipour, Artadokht
Malhotra, Jyoti
Ferretti, Patrizia
Phadke, Rahul
Muntoni, Francesco
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Article History
Received: 27 December 2024
Accepted: 2 April 2025
First Online: 21 May 2025
Declarations
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: Human tissue procedures were conducted in accordance with the regulations with informed consent in accordance with the UK Human Tissue Act 2006 for study participation under ethical approval (NRES Committee London—Fulham, UK; REC: 18/LO/0822). The human embryonic and foetal material was provided by the Joint MRC/Wellcome Trust (Grants MR/006237/1, MR/X008304/1 and 226202/Z/22/Z) Human Developmental Biology Resource (). Adult human brains were selected and procured from the Edinburgh Brain Bank (Ethic Ref 20/PR/0582). Tissue samples were obtained from the Edinburgh Brain Bank (EBB) and BRAIN UK, which is supported by Brain Tumour Research and has been established with the support of the British Neuropathological Society and the Medical Research Council.
: Not applicable.
: As COI, FM is an investigator in Sarepta, Genethon, Roche clinical trials. He is the PI of the Sarepta grant supporting this work (funding to UCL), and has participated to advisory boards and/or symposia for Sarepta, Roche, Dyne therapeutics, Wave and Entrada.
