Emerging Therapies in β-Thalassemia

Advances in understanding the underlying pathophysiology of β-thalassemia have enabled efforts toward the development of novel therapeutic modalities . These can be classified into three major categories based on their ability to target different features of the underlying disease pathophysiology: correction of the α/β globin chain imbalance, targeting ineffective erythropoiesis , and targeting iron dysregulation. This article provides an overview of these different emerging therapies that are currently in development for β-thalassemia.