Painful vaso-occlusive crisis (VOC) remains the most common reason for presenting to the
Emergency Department and hospitalization in patients with sickle cell disease (SCD) …

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 …

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

Hemoglobinopathies, including thalassemias and sickle cell disease, are the most common
monogenic diseases worldwide, with estimated annual births of more than 330,000 affected …

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A
phase 2, open-label study Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in …

Mutations in the PKLR gene lead to pyruvate kinase (PK) deficiency, causing chronic
hemolytic anemia secondary to reduced red cell energy, which is crucial for maintenance of …

Sickle cell disease is the most common inherited pathological haemoglobinopathy. Over the
past 30 years, disease-related morbidity and mortality have improved in high-income …

This article provides an overview of conventional, new, and future treatment options for
sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current …

Targeting the primary pathogenic event of sickle cell disease (SCD), the polymerization of
sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral …

Polymerization of deoxygenated sickle hemoglobin (HbS) leads to erythrocyte sickling.
Enhancing activity of the erythrocyte glycolytic pathway has anti-sickling potential as this …