Mitapivat, a novel pyruvate kinase activator, for the treatment of hereditary hemolytic anemias
H Al-Samkari, EJ van Beers - Therapeutic advances in …, 2021 - journals.sagepub.com
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 …
pyruvate kinase enzyme. Mitapivat has been shown to significantly upregulate both wild …
[HTML][HTML] Molecular heterogeneity of pyruvate kinase deficiency
P Bianchi, E Fermo - Haematologica, 2020 - ncbi.nlm.nih.gov
Red cell pyruvate kinase (PK) deficiency is the most common glycolytic defect associated
with congenital non-spherocytic hemolytic anemia. The disease, transmitted as an …
with congenital non-spherocytic hemolytic anemia. The disease, transmitted as an …
Mitapivat versus placebo for pyruvate kinase deficiency
H Al-Samkari, F Galactéros, A Glenthøj… - … England Journal of …, 2022 - Mass Medical Soc
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 …
associated with hemolytic anemia. In a phase 2 study, mitapivat, an oral, first-in-class …
Updates and advances in pyruvate kinase deficiency
N Luke, K Hillier, H Al-Samkari, RF Grace - Trends in molecular medicine, 2023 - cell.com
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 …
hemolytic anemia secondary to reduced red cell energy, which is crucial for maintenance of …
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency
H Al-Samkari, EJ van Beers, DH Morton… - Blood …, 2022 - ashpublications.org
Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic
hemolytic anemia. Although recognition of the disease spectrum has recently expanded …
hemolytic anemia. Although recognition of the disease spectrum has recently expanded …
Identification of metabolic biomarkers in relation to methotrexate response in early rheumatoid arthritis
HR Gosselt, IB Muller, G Jansen… - Journal of personalized …, 2020 - mdpi.com
This study aimed to identify baseline metabolic biomarkers for response to methotrexate
(MTX) therapy in rheumatoid arthritis (RA) using an untargeted method. In total, 82 baseline …
(MTX) therapy in rheumatoid arthritis (RA) using an untargeted method. In total, 82 baseline …
Pyruvate kinase activators: targeting red cell metabolism in sickle cell disease
JZ Xu, GM Vercellotti - Hematology, 2023 - ashpublications.org
Hemoglobin S (HbS) polymerization, red blood cell (RBC) sickling, chronic anemia, and
vaso-occlusion are core to sickle cell disease (SCD) pathophysiology. Pyruvate kinase (PK) …
vaso-occlusion are core to sickle cell disease (SCD) pathophysiology. Pyruvate kinase (PK) …
Mitapivat: A Review in Pyruvate Kinase Deficiency in Adults
A Zhuang-Yan, M Shirley - Drugs, 2023 - Springer
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 …
USA for the treatment of haemolytic anaemia in adults with PK deficiency and in the EU and …
The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency
H Al‐Samkari, K Addonizio, B Glader… - British Journal of …, 2021 - Wiley Online Library
Diagnosis of pyruvate kinase deficiency (PKD), the most common cause of hereditary non‐
spherocytic haemolytic anaemia, remains challenging in routine practice and no biomarkers …
spherocytic haemolytic anaemia, remains challenging in routine practice and no biomarkers …
[HTML][HTML] Evaluation of Glucose 6-Phosphate Dehydrogenase, Pyruvate Kinase, and New Generation Inflammation Biomarkers in Prolonged Neonatal Jaundice
Background and Objectives: To evaluate the clinical findings of glucose 6-phosphate
dehydrogenase (G6PD) and pyruvate kinase (PK) deficiency in prolonged jaundice and to …
dehydrogenase (G6PD) and pyruvate kinase (PK) deficiency in prolonged jaundice and to …