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Significant Improvements in Survival and Developmental Milestones Demonstrated in Type 1 SMA Patients Treated with Evrysdi™

Significant Improvements in Survival and Developmental Milestones Demonstrated in Type 1 SMA Patients Treated with Evrysdi™
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Significant Improvements in Survival and Developmental Milestones Demonstrated in Type 1 SMA Patients Treated with Evrysdi™

Significant Improvements in Survival and Developmental Milestones Demonstrated in Type 1 SMA Patients Treated with Evrysdi™
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Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker Muscular Dystrophy

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

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

Italfarmaco Group: Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker Muscular Dystrophy
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Evrysdi™ Approved in Japan for the Treatment of Spinal Muscular Atrophy

Evrysdi™ Approved in Japan for the Treatment of Spinal Muscular Atrophy
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