One hundred years have passed since Warburg discovered alterations in cancer
metabolism, more than 70 years since Sidney Farber introduced anti-folates that …

Cancer has myriad effects on metabolism that include both rewiring of intracellular
metabolism to enable cancer cells to proliferate inappropriately and adapt to the tumor …

Background Pyruvate kinase deficiency is a rare, hereditary, chronic condition that is
associated with hemolytic anemia. In a phase 2 study, mitapivat, an oral, first-in-class …

Background Patients with non-transfusion-dependent thalassaemia (NTDT), although they
do not require regular blood transfusions for survival, can still accrue a heavy burden of …

Polymerization of deoxygenated hemoglobin S underlies the pathophysiology of sickle cell
disease (SCD). In activating red blood cell pyruvate kinase and glycolysis, mitapivat (AG …

The erythroid marrow and circulating red blood cells (RBCs) are the key components of the
human erythron. Abnormalities of the erythron that are responsible for anemia can be …

The treatment landscape for patients with β‐thalassemia is witnessing a swift evolution, yet
several unmet needs continue to persist. Patients with transfusion‐dependent β …

Mitapivat (AG-348) is a novel, first-in-class oral small molecule allosteric activator of the
pyruvate kinase enzyme. Mitapivat has been shown to significantly upregulate both wild …

Anemia in β-thalassemia is related to ineffective erythropoiesis and reduced red cell
survival. Excess free heme and accumulation of unpaired α-globin chains impose …

Pyruvate kinase deficiency (PKD) is an autosomal-recessive enzyme defect of the glycolytic
pathway that causes congenital nonspherocytic hemolytic anemia. The diagnosis and …