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In March 2024, the US FDA approved Italfarmaco's givinostat (Duvyzat) for Duchenne muscular dystrophy (DMD) patients aged six and older. In this video, we'll break down what Givinostat is, how it works, and the results of the clinical trial that has led to its approval.
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References:
1. Venugopal V, Pavlakis S. Duchenne Muscular Dystrophy. In: StatPearls. Treasure Island (FL): StatPearls Publishing; July 10, 2023.
2. Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial [published correction appears in Lancet Neurol. 2024 Apr 18;:]. Lancet Neurol. 2024;23(4):393-403. doi:10.1016/S1474-4422(24)00036-X
3. Mozzetta C, Sartorelli V, Steinkuhler C, Puri PL. HDAC inhibitors as pharmacological treatment for Duchenne muscular dystrophy: a discovery journey from bench to patients [published correction appears in Trends Mol Med. 2024 Apr 19;:]. Trends Mol Med. 2024;30(3):278-294. doi:10.1016/j.molmed.2024.01.007
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