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Papers of the Week

Home / Publications / Pain Research Forum / Papers of the Week / Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.

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2019 Sep 05


N Engl J Med


http://www.ncbi.nlm.nih.gov/pubmed/31483964?dopt=Abstract


381


10

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.

Authors

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.
Grace RF, Rose C, Layton MD, Galactéros F, Barcellini W, Morton HD, van Beers EJ, Yaish H, Ravindranath Y, Kuo KHM, Sheth S, Kwiatkowski JL, Barbier AJ, Bodie S, Silver B, Hua L, Kung C, Hawkins P, Jouvin M-H, Bowden C, et al.
N Engl J Med. 2019 Sep 05; 381(10):933-944.
PMID: 31483964.

Abstract

Pyruvate kinase deficiency is caused by mutations in and leads to congenital hemolytic anemia. Mitapivat is an oral, small-molecule allosteric activator of pyruvate kinase in red cells.
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