Article
AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes.
Haematologica - 1 Jan 2021
Rab Minke A E, Van Oirschot Brigitte A, Kosinski Penelope A, Hixon Jeffrey, Johnson Kendall, Chubukov Victor, Dang Lenny, Pasterkamp Gerard, Van Straaten Stephanie, Van Solinge Wouter W, Van Beers Eduard J, Kung Charles, Van Wijk Richard
Abstract excerpt
Pyruvate kinase (PK) deficiency is a rare hereditary disorder affecting red cell (RBC) glycolysis, causing changes in metabolism including a deficiency in ATP. This affects red cell homeostasis, promoting premature removal of RBCs from the circulation. In this study we characterized and evaluated the effect of AG-348, an allosteric activator of PK that is currently in clinical trials for treatment of PK...
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