Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study

OBJECTIVE: No treatments are approved for Becker muscular dystrophy (BMD). This study investigated the efficacy and safety of givinostat, a histone deacetylase pan-inhibitor, in adults with BMD. METHODS: Males aged 18–65 years with a diagnosis of BMD confirmed by genetic testing were randomized 2:1...

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Detalles Bibliográficos
Autores principales: Comi, Giacomo P., Niks, Erik H., Vandenborne, Krista, Cinnante, Claudia M., Kan, Hermien E., Willcocks, Rebecca J., Velardo, Daniele, Magri, Francesca, Ripolone, Michela, van Benthem, Jules J., van de Velde, Nienke M., Nava, Simone, Ambrosoli, Laura, Cazzaniga, Sara, Bettica, Paolo U.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Neurology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9923355/
https://www.ncbi.nlm.nih.gov/pubmed/36793492
http://dx.doi.org/10.3389/fneur.2023.1095121

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