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

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

ObjectiveNo 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.MethodsMales aged 18–65 years with a diagnosis of BMD confirmed by genetic testing were randomized 2:1 to 12...

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Bibliographic Details
Main Authors: Giacomo P. Comi, Erik H. Niks, Krista Vandenborne, Claudia M. Cinnante, Hermien E. Kan, Rebecca J. Willcocks, Daniele Velardo, Francesca Magri, Michela Ripolone, Jules J. van Benthem, Nienke M. van de Velde, Simone Nava, Laura Ambrosoli, Sara Cazzaniga, Paolo U. Bettica
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-01-01
Series:Frontiers in Neurology
Subjects:
Becker muscular dystrophy
therapy
disease progression
fibrosis
magnetic resonance imaging (MRI)
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2023.1095121/full

Internet

https://www.frontiersin.org/articles/10.3389/fneur.2023.1095121/full

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