Emerging Therapies in β-Thalassemia

Abstract

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.

Keywords: Advancement of care; Clinical trial; Novel therapies; Quality of life; Transfusion burden; β-Thalassemia.