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In Vivo Studies of a Novel HDAC Inhibitor for Treating Hemoglobin DisordersSBC: Cetya Therapeutics, Inc. Topic: NHLBI
PROJECT SUMMARY The β-hemoglobinopathies are prevalent genetic blood diseases with few treatment options. It is estimate that 7% of the worldandapos;s population carries an abnormal hemoglobin gene with 400,000 infants born annually with a severe life threatening hemoglobinopathy. Drug mediated induction of normal, but developmentally silenced, fetal hemoglobin (HbF) expression reduces anemia and ...STTR Phase II 2019 Department of Health and Human ServicesNational Institutes of Health