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Posted on 8181 Italfarmaco Group announced today topline data from its proof-of-concept Phase 2 trial with Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor, in 51 adult males with Becker Muscular Dystrophy (BMD). The study was designed to evaluate the effect of Givinostat in BMD, building on the experience developed in Duchenne Muscular Dystrophy. Based on this experience, change in total fibrosis in the muscle biopsy was selected as the primary endpoint of the study and change in fat fraction and contractile CSA using quantitative Magnetic Resonance Imaging (MRI) as key secondary endpoints. Givinostat did not show significant difference in the primary endpoint compared to placebo. However, significant difference from placebo in MRI of muscles in the whole thigh as well as quadriceps confirmed the ability of Givinostat to counteract muscle deterioration in the treated BMD patients. The safety

San diegoUnited statesRegione lombardiaPaolo betticaKrista vandenborneEuropean regional development fundSanta lucia foundationUniversity of floridaDepartment of physical therapyRegional operational programItalfarmaco groupNeurologist at leiden university medical centreUniversity of milanBecker muscular dystrophyDuchenne muscularMagnetic resonance imaging