Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study.
Comi GP, Niks EH, Vandenborne K, Cinnante CM, Kan HE, Willcocks RJ, Velardo D, Magri F, Ripolone M, van Benthem JJ, van de Velde NM, Nava S, Ambrosoli L, Cazzaniga S, Bettica PU.
Comi GP, et al.
Front Neurol. 2023 Jan 30;14:1095121. doi: 10.3389/fneur.2023.1095121. eCollection 2023.
Front Neurol. 2023.
PMID: 36793492
Free PMC article.
METHODS: Males aged 18-65 years with a diagnosis of BMD confirmed by genetic testing were randomized 2:1 to 12 months treatment with givinostat or placebo. The primary objective was to demonstrate statistical superiority of givinostat over placebo for mean change fr …
METHODS: Males aged 18-65 years with a diagnosis of BMD confirmed by genetic testing were randomized 2:1 to 12 months treatment with givi …