Results 111 to 120 of about 405 (131)
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Safety and Efficacy of Mitapivat in Adult Patients with Erythrocyte Membranopathies (SATISFY)
Blood, 2023Background: Membranopathies encompass hemolytic disorders arising from genetic defects in erythrocyte membrane proteins and include hereditary spherocytosis and stomatocytosis. Congenital dyserythropoietic anemia type II (CDA II) is associated with SEC23B gene variants and its phenotype may be similar to hereditary ...
Andreas Glenthoej +12 more
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Mitapivat: a step forward across different hemolytic diseases
Expert Opinion on Emerging DrugsAli Taher, Nicole Charbel
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American Journal of Therapeutics, 2023
Background: Pyruvate kinase (PK) deficiency is a rare enzyme-linked glycolytic defect resulting in mild-to-severe chronic persistent erythrocyte hemolysis. The disease is an autosomal recessive trait caused by mutations in the PK liver and red blood cell gene characterized by insufficient erythrocyte PK activity.
Josef Wills +4 more
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Background: Pyruvate kinase (PK) deficiency is a rare enzyme-linked glycolytic defect resulting in mild-to-severe chronic persistent erythrocyte hemolysis. The disease is an autosomal recessive trait caused by mutations in the PK liver and red blood cell gene characterized by insufficient erythrocyte PK activity.
Josef Wills +4 more
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An Alternate, Efficient Synthetic Process for a Hemolytic Anemia Drug: Mitapivat Sulfate
Organic Process Research and DevelopmentSurendrababu M S, Naresh Kumar Katari
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Mitapivat: A Novel Drug Discovery for the Treatment of Hereditary Hemolytic Anaemias
Current Indian Science: 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-type and numerous mutant forms of erythrocyte pyruvate kinase (PKR), increasing adenosine triphosphate (ATP) production and ...
Shikha Sharma, Utkarsh Sharma
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Mitapivat for treatment of pyruvate kinase deficiency
The Lancet Haematology, 2022openaire +2 more sources
Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency
Current Medicinal ChemistryAbstract: Mitapivat is an orally bioavailable small molecule allosteric activator of pyruvate kinase. It was approved by the US FDA on February 17, 2022, and the European Union in November 2022 for the treatment of hemolytic anemia in adult patients with pyruvate kinase deficiency.
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Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency
Current Medicinal Chemistry, 2023Surya K De
exaly
Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency
Blood, 2022Eduard J. van Beers +11 more
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