Novel Therapeutic Approaches in Thalassemias, Sickle Cell Disease and Other Red Cell Disorders
Valeria Maria Pinto,
Filippo Mazzi,
Lucia De Franceschi
Abstract:In this last decade, a deeper understanding of the pathophysiology of hereditary red cell disorders and the development of novel classes of pharmacologic agents have provided novel therapeutic approaches to thalassemias, sickle cell disease (SCD) and other red cell disorders. Here, we analyze and discuss the novel therapeutic options according to their targets and taking into consideration the complex process of erythroid differentiation, maturation, and survival of erythrocytes in the peripheral circulation. … Show more
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