Articles with "mitapivat" as a keyword



Relative Bioavailability Studies With Mitapivat: Formulation and Food Effect Assessments in Healthy Subjects

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Published in 2024 at "Clinical Pharmacology in Drug Development"

DOI: 10.1002/cpdd.1481

Abstract: Pyruvate kinase (PK) deficiency is a rare, hereditary, hemolytic anemia caused by mutations in the PKLR gene encoding the PK enzyme. Mitapivat (previously designated AG‐348) is a first‐in‐class, oral, allosteric activator of PK. We report… read more here.

Keywords: relative bioavailability; food; mitapivat; bioavailability studies ... See more keywords

Long-term mitapivat treatment improves inflammatory pro-fibrotic cardiomyopathy in a murine model of β-thalassemia.

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Published in 2025 at "Blood"

DOI: 10.1182/blood-2025-727

Abstract: Hypertrophic cardiomyopathy (HC) is a severe invalidating complication of ß–thalassemia (ß–thal), contributing to reduced patients' life expectancy. Recently we showed that 12-months old male ß-thal mice develop HC independently from heart iron accumulation, and that… read more here.

Keywords: mice; mitapivat; pro fibrotic; heart ... See more keywords