An evaluation of mitapivat for the treatment of non-transfusion-dependent alpha- and beta-thalassemia.
Non-transfusion-dependent thalassemia (NTDT) describes a group of inherited hemoglobin disorders characterized by hemolytic anemia and ineffective erythropoiesis. Although patients with NTDT do not require lifelong regular transfusions for survival, they are at a substantial risk of complications including iron overloa...