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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 ....

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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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Novartis Pharma AG: New Zolgensma data demonstrate age-appropriate development when used presymptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline

Novartis Pharma AG: New Zolgensma data demonstrate age-appropriate development when used presymptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline
finanznachrichten.de - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from finanznachrichten.de Daily Mail and Mail on Sunday newspapers.

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Biogen Inc.: New Data at Cure SMA 2021 Highlight the Long-Term Efficacy of SPINRAZA (nusinersen) and Biogen's Commitment to Innovation in SMA Therapy

New data analysis suggests an investigational higher dose of SPINRAZA may lead to clinically meaningful improvements in motor functionA NURTURE study analysis shows 92 percent of children who initiated ....

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New Genentech Data at 2021 AAN Highlight Impact and Breadth of Expanding Neuroscience Portfolio

Press release content from Business Wire. The AP news staff was not involved in its creation.
New Genentech Data at 2021 AAN Highlight Impact and Breadth of Expanding Neuroscience Portfolio
April 8, 2021 GMT
SOUTH SAN FRANCISCO, Calif. (BUSINESS WIRE) Apr 8, 2021
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that new data for its approved and investigational medicines for the treatment of neurological disorders will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021. These new data include 23 abstracts highlighting the expanding Genentech neuroscience portfolio across six therapeutic areas, including Evrysdi™ (risdiplam) for spinal muscular atrophy (SMA), Ocrevus ® (ocrelizumab) in relapsing and primary progressive multiple sclerosis (RMS and PPMS), investigational Bruton’s tyrosine kinase inhibitor (BTKi) fenebrutinib in Phase III trials for RMS and PPMS, E ....

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