Therapeutic options for rare congenital (hemoglobinopathies, membrane and enzyme
defects, congenital dyserythropoietic anemia) and acquired anemias [warm autoimmune …

Significance: Sickle cell disease (SCD) is the most common inherited diathesis affecting
mostly underserved populations globally. SCD is characterized by chronic pain and fatigue …

Introduction Membranopathies encompass haemolytic disorders arising from genetic
variants in erythrocyte membrane proteins, including hereditary spherocytosis and …

Preclinical and clinical evidence indicate that pyruvate kinase activators represent new
therapeutic option in hemoglobinopathies or SCD. Other red cell disorders such as …

Pyruvate kinase (PK) is a key enzyme in glycolysis, the sole source of adenosine
triphosphate, which is essential for all energy-dependent activities of red blood cells …

Mitapivat (Pyrukynd®), an oral, allosteric activator of pyruvate kinase (PK), is approved in the
USA for the treatment of haemolytic anaemia in adults with PK deficiency and in the EU and …

Mitapivat, a pyruvate kinase activator, shows great potential as a sickle cell disease (SCD)-
modifying therapy. The safety and efficacy of mitapivat as a long-term maintenance therapy …

Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency Early-onset
reduced bone mineral density in patients with pyruvate kinase deficiency Am J Hematol. 2023 …

Pyruvate kinase M2 (PKM2), a rate limiting enzyme in glycolysis, is a cellular regulator that
has received extensive attention and regards as a metabolic regulator of cellular metabolism …

Introduction Pyruvate kinase (PK) deficiency is a rare, under-recognised, hereditary
condition that leads to chronic haemolytic anaemia and potentially serious secondary …