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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 ....

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 ,

Tumor marker may help overcome endocrine treatment-resistant breast cancer


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Credit: Sanford Burnham Prebys
LA JOLLA, CALIF. - May 19, 2021 - A study led by scientists at Sanford Burnham Prebys Medical Discovery Institute has identified a tumor marker that may be used to predict which breast cancer patients will experience resistance to endocrine therapy. The research offers a new approach to selecting patients for therapy that targets HER2, a protein that promotes the growth of cancer cells, to help avoid disease relapse or progression of endocrine-sensitive disease.
The study was published in the journal
Nature Communications.
Nearly 80% of breast tumors are estrogen receptor (ER)-positive. For decades, these tumors have been treated with anti-estrogen (endocrine) therapies to lower estrogen levels and help slow the cancer s growth. About 20% of breast cancers are also HER2-positive at diagnosis, and these tumors tend to be more aggressive and fast-growing, as HER2 is a receptor that when active, promotes the rapid growth of ....

United States , Nindo Punturi , Shunqiang Li , Vaishnavi Devarakonda , Susang Komen , Svasti Haricharan , Shyamm Kavuri , Ching Hui Chen , Rashi Kalra , Nindob Punturi , Sinem Seker , Sanford Burnham Prebys , Matthewj Ellis , Nature Communications , National Cancer Institute , Program At Sanford Burnham Prebys , Alliance For Clinical Trials In Oncology , National Institutes Of Health , Cancer Center , Sanford Burnham Prebys Medical Discovery Institute , Tina Primeau At Washington University In St , Neozenome Therapeutics Inc , Career Development , Sanford Burnham Prebys Medical Research Institute , Immuno Oncology Program , Aloran Mazumder ,