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UF Team Crucial to New Duchenne Muscular Dystrophy Drug Approval

UF Team Crucial to New Duchenne Muscular Dystrophy Drug Approval
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Krista Vandenborne , Drug Administration , University Of Florida , Health Professions , Uf College Of Public Health , Lancet Neurology , Public Health ,

UF team plays major role in success of newly approved Duchenne muscular dystrophy drug

UF team plays major role in success of newly approved Duchenne muscular dystrophy drug
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Krista Vandenborne , Uf College Of Public Health , Drug Administration , University Of Florida , Health Professionsand , Lancet Neurology , Public Health ,

Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker Muscular Dystrophy


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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. Th ....

San Diego , United States , Regione Lombardia , Paolo Bettica , Krista Vandenborne , European Regional Development Fund , Santa Lucia Foundation , University Of Florida , Department Of Physical Therapy , Regional Operational Program , Italfarmaco Group , Neurologist At Leiden University Medical Centre , University Of Milan , Becker Muscular Dystrophy , Duchenne Muscular , Magnetic Resonance Imaging , Parent Project Muscular Dystrophy , Muscular Dystrophy , Chief Medical Officer , Duchenne Muscular Dystrophy , Transfer Domain , Motor Function Measure , Adverse Events , Treatment Emergent Adverse Events , Principal Investigator , Physical Therapy ,