Abstract
Sickle cell disease (SCD) affects millions of people throughout the world. Hemoglobin S (HbS) polymerization is the fundamental cause of SCD pathophysiology, which leads to hemolysis, increased viscosity, and acute vaso-occlusive episodes. Novel agents have been developed to target the pathophysiology of SCD and decrease the frequency of SCD complications. Voxelotor (Oxbryta) is an HbS polymerization inhibitor that is approved by the U.S. Food & Drug Administration for the treatment of SCD in adults and pediatric patients 12 years and older.
