Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency

•Mitapivat was associated with meaningful long-term improvements in key markers of iron homeostasis and erythropoiesis.•Mitapivat is the first disease-modifying pharmacotherapy to improve iron overload in adult patients with PK deficiency. [Display omitted] Pyruvate kinase (PK) deficiency is a rare,...

Ausführliche Beschreibung