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Acipimox in Mitochondrial Myopathy (AIMM): study protocol for a randomised, double-blinded, placebo-controlled, adaptive design trial of the efficacy of acipimox in adult patients with mitochondrial myopathy

BACKGROUND: Mitochondrial disease is a heterogenous group of rare, complex neurometabolic disorders. Despite their individual rarity, collectively mitochondrial diseases represent the most common cause of inherited metabolic disorders in the UK; they affect 1 in every 4300 individuals, up to 15,000...

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Detalles Bibliográficos
Autores principales: Abouhajar, Alaa, Alcock, Lisa, Bigirumurame, Theophile, Bradley, Penny, Brown, Laura, Campbell, Ian, Del Din, Sylvia, Faitg, Julie, Falkous, Gavin, Gorman, Gráinne S., Lakey, Rachel, McFarland, Robert, Newman, Jane, Rochester, Lynn, Ryan, Vicky, Smith, Hesther, Steel, Alison, Stefanetti, Renae J., Su, Huizhong, Taylor, Robert W., Thomas, Naomi J.P., Tuppen, Helen, Vincent, Amy E., Warren, Charlotte, Watson, Gillian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9486776/
https://www.ncbi.nlm.nih.gov/pubmed/36127727
http://dx.doi.org/10.1186/s13063-022-06544-x