Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
About this item
Full title
Author / Creator
Grace, Rachael F , Rose, Christian , Layton, D. Mark , Galactéros, Frédéric , Barcellini, Wilma , Morton, D. Holmes , van Beers, Eduard J , Yaish, Hassan , Ravindranath, Yaddanapudi , Kuo, Kevin H.M , Sheth, Sujit , Kwiatkowski, Janet L , Barbier, Ann J , Bodie, Susan , Silver, Bruce , Hua, Lei , Kung, Charles , Hawkins, Peter , Jouvin, Marie-Hélène , Bowden, Chris and Glader, Bertil
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
In this study, approximately half the patients with red-cell pyruvate kinase deficiency who were treated with mitapivat had an improvement in their hemoglobin level and decreased hemolysis that was sustained for nearly 3 years. Patients who had missense mutations that allowed for synthesis of a hypofunctioning enzyme were most likely to have a resp...
Alternative Titles
Full title
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
Authors, Artists and Contributors
Author / Creator
Rose, Christian
Layton, D. Mark
Galactéros, Frédéric
Barcellini, Wilma
Morton, D. Holmes
van Beers, Eduard J
Yaish, Hassan
Ravindranath, Yaddanapudi
Kuo, Kevin H.M
Sheth, Sujit
Kwiatkowski, Janet L
Barbier, Ann J
Bodie, Susan
Silver, Bruce
Hua, Lei
Kung, Charles
Hawkins, Peter
Jouvin, Marie-Hélène
Bowden, Chris
Glader, Bertil
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2285104036
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2285104036
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1902678
