Mitapivat Improves Ineffective Erythropoiesis and Iron Overload in Adult Patients with Pyruvate Kinase Deficiency

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Mitapivat’s impact on iron overload and ineffective erythropoiesis was evaluated in adults with pyruvate kinase deficiency who were not regularly transfused in the Phase III ACTIVATE trial and long-term extension.
[Blood Advances]
Abstract