Doubling down on PK activation for sickle cell disease.
作者信息
Wilson Samuel R, Pecker Lydia H
机构信息
Department of Medicine, Hematology and Blood Research Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC.
Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD.
出版信息
Blood Adv. 2024 Aug 27;8(16):4457-4458. doi: 10.1182/bloodadvances.2024013367.
Abstract
摘要
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本文引用的文献
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Multicenter, phase 1 study of etavopivat (FT-4202) treatment for up to 12 weeks in patients with sickle cell disease.多中心、1 期研究评估 etavopivat（FT-4202）治疗镰状细胞病患者长达 12 周的疗效。
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Recent developments in the use of pyruvate kinase activators as a new approach for treating sickle cell disease.丙酮酸激酶激活剂在治疗镰状细胞病中的新应用研究进展。
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Am J Hematol. 2023 Jul;98(7):1087-1096. doi: 10.1002/ajh.26956. Epub 2023 May 12.
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A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease.一项关于丙酮酸激酶激活剂米替泊肟（AG-348）在镰状细胞病中的 1 期剂量递增研究。
Blood. 2022 Nov 10;140(19):2053-2062. doi: 10.1182/blood.2022015403.
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J Pharmacol Exp Ther. 2022 Mar;380(3):210-219. doi: 10.1124/jpet.121.000743. Epub 2022 Jan 14.
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Lancet Haematol. 2021 May;8(5):e323-e333. doi: 10.1016/S2352-3026(21)00059-4. Epub 2021 Apr 7.
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A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.一项针对镰状细胞病患者的 voxotor 的 3 期随机试验。
N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.
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