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

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

Frontiers in neurology（2023）

引用 6|浏览20

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摘要

The study failed to achieve the primary endpoint. However, there was a potential signal from the MRI assessments suggesting givinostat could prevent (or slow down) BMD disease progression.

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关键词

Becker muscular dystrophy,disease progression,fibrosis,magnetic resonance imaging (MRI),therapy

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