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American University
Major advancements in transfusion programs and iron chelation therapy have significantly increased the life expectancy and quality of life for beta-thalassemia patients. This article discusses various emerging therapies aimed at correcting the underlying disease pathophysiology, including approaches to address globin chain imbalance, ineffective erythropoiesis, and iron dysregulation. The goal of these therapies is to enhance patient compliance, reduce symptom burden, and ultimately improve health outcomes for those affected by beta-thalassemia.
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