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 activator of erythrocyte pyruvate ...
Pyruvate kinase deficiency is caused by mutations in PKLR and leads to congenital hemolytic anemia. Mitapivat is an oral, small-molecule allosteric activator of pyruvate kinase in red cells. Common ...
The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application for SL1009 (sodium dichloroacetate oral solution) for the treatment of pyruvate dehydrogenase complex ...