Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia.
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DOI: 10.1089/hum.2023.020
Abstract: Friedreich's ataxia (FA) is a life-threatening autosomal recessive disorder characterized by neurologic and cardiac dysfunction. Arrythmias and heart failure are the main cause of premature death. From prior studies in murine models of FA, AAV… read more here.
Keywords: dose aavrh; dose; cardiac manifestations; friedreich ataxia ... See more keywords