Mitapivat versus Placebo for Pyruvate Kinase Deficiency
Mitapivat versus Placebo for Pyruvate Kinase Deficiency
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Author / Creator
ACTIVATE Investigators , Al-Samkari, Hanny , Galactéros, Frédéric , Glenthøj, Andreas , Rothman, Jennifer A , Andres, Oliver , Grace, Rachael F , Morado-Arias, Marta , Layton, D. Mark , Onodera, Koichi , Verhovsek, Madeleine , Barcellini, Wilma , Chonat, Satheesh , Judge, Malia P , Zagadailov, Erin , Xu, Rengyi , Hawkins, Peter , Beynon, Vanessa , Gheuens, Sarah and van Beers, Eduard J
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
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Scope and Contents
Contents
Pyruvate kinase deficiency, the most common genetic lesion in the glycolytic pathway, leads to chronic hemolytic anemia. Mitapivat, an oral agent, can activate some mutant enzymes and restore red-cell ATP generation. In this trial, hemoglobin levels increased from baseline by 1.5 g per deciliter or more at 24 weeks in 40% of the patients with pyruv...
Alternative Titles
Full title
Mitapivat versus Placebo for Pyruvate Kinase Deficiency
Authors, Artists and Contributors
Author / Creator
Al-Samkari, Hanny
Galactéros, Frédéric
Glenthøj, Andreas
Rothman, Jennifer A
Andres, Oliver
Grace, Rachael F
Morado-Arias, Marta
Layton, D. Mark
Onodera, Koichi
Verhovsek, Madeleine
Barcellini, Wilma
Chonat, Satheesh
Judge, Malia P
Zagadailov, Erin
Xu, Rengyi
Hawkins, Peter
Beynon, Vanessa
Gheuens, Sarah
van Beers, Eduard J
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2650252539
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2650252539
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2116634
