Mitapivat for phosphofructokinase deficiency.
作者信息
Al-Samkari Hanny
机构信息
Division of Hematology Oncology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
出版信息
Am J Hematol. 2024 Oct;99(10):2045-2046. doi: 10.1002/ajh.27442. Epub 2024 Jul 23.
Abstract
摘要
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本文引用的文献
1
Mitapivat versus Placebo for Pyruvate Kinase Deficiency.
N Engl J Med. 2022 Apr 14;386(15):1432-1442. doi: 10.1056/NEJMoa2116634.
2
Mitapivat, a novel pyruvate kinase activator, for the treatment of hereditary hemolytic anemias.
Ther Adv Hematol. 2021 Dec 21;12:20406207211066070. doi: 10.1177/20406207211066070. eCollection 2021.
3
Phosphofructokinase deficiency; past, present and future.
Curr Mol Med. 2002 Mar;2(2):197-212. doi: 10.2174/1566524024605734.
4
The RAND 36-Item Health Survey 1.0.
Health Econ. 1993 Oct;2(3):217-27. doi: 10.1002/hec.4730020305.
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