Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia

Kuo and colleagues(1) evaluated the safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion-dependent α-thalassemia or β-thalassemia. The high rate of hemoglobin response and good tolerability encourages further development in thalassemia.