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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…
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Keywords:
relative bioavailability;
food;
mitapivat;
bioavailability studies ... See more keywords
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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…
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Keywords:
mice;
mitapivat;
pro fibrotic;
heart ... See more keywords