I am a
Home I AM A Search Login

Papers of the Week

2019 Sep 05

N Engl J Med



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.


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.