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Page 1
Mitapivat increases ATP and decreases oxidative stress and erythrocyte mitochondria retention in a SCD mouse model.
Quezado ZMN, Kamimura S, Smith M, Wang X, Heaven MR, Jana S, Vogel S, Zerfas P, Combs CA, Almeida LEF, Li Q, Quezado M, Horkayne-Szakaly I, Kosinski PA, Yu S, Kapadnis U, Kung C, Dang L, Wakim P, Eaton WA, Alayash AI, Thein SL. Quezado ZMN, et al. Among authors: kosinski pa. Blood Cells Mol Dis. 2022 Jul;95:102660. doi: 10.1016/j.bcmd.2022.102660. Epub 2022 Mar 12. Blood Cells Mol Dis. 2022. PMID: 35366607 Free PMC article.
AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency.
Kung C, Hixon J, Kosinski PA, Cianchetta G, Histen G, Chen Y, Hill C, Gross S, Si Y, Johnson K, DeLaBarre B, Luo Z, Gu Z, Yao G, Tang H, Fang C, Xu Y, Lv X, Biller S, Su SM, Yang H, Popovici-Muller J, Salituro F, Silverman L, Dang L. Kung C, et al. Among authors: kosinski pa. Blood. 2017 Sep 14;130(11):1347-1356. doi: 10.1182/blood-2016-11-753525. Epub 2017 Jul 31. Blood. 2017. PMID: 28760888 Free PMC article.
AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes.
Rab MAE, Van Oirschot BA, Kosinski PA, Hixon J, Johnson K, Chubukov V, Dang L, Pasterkamp G, Van Straaten S, Van Solinge WW, Van Beers EJ, Kung C, Van Wijk R. Rab MAE, et al. Among authors: kosinski pa. Haematologica. 2021 Jan 1;106(1):238-249. doi: 10.3324/haematol.2019.238865. Haematologica. 2021. PMID: 31974203 Free PMC article.
Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy.
Rab MAE, Bos J, van Oirschot BA, van Straaten S, Kosinski PA, Chubukov V, Kim H, Mangus H, Schutgens REG, Pasterkamp G, Dang L, Kung C, van Beers EJ, van Wijk R. Rab MAE, et al. Among authors: kosinski pa. Blood. 2021 May 27;137(21):2997-3001. doi: 10.1182/blood.2020008635. Blood. 2021. PMID: 33690814 Free PMC article. No abstract available.
A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease.
Xu JZ, Conrey A, Frey I, Gwaabe E, Menapace LA, Tumburu L, Lundt M, Lequang T, Li Q, Glass K, Dunkelberger EB, Iyer V, Mangus H, Kung C, Dang L, Kosinski PA, Hawkins P, Jeffries N, Eaton WA, Lay Thein S. Xu JZ, et al. Among authors: kosinski pa. Blood. 2022 Nov 10;140(19):2053-2062. doi: 10.1182/blood.2022015403. Blood. 2022. PMID: 35576529 Free PMC article. Clinical Trial.
The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model.
Matte A, Federti E, Kung C, Kosinski PA, Narayanaswamy R, Russo R, Federico G, Carlomagno F, Desbats MA, Salviati L, Leboeuf C, Valenti MT, Turrini F, Janin A, Yu S, Beneduce E, Ronseaux S, Iatcenko I, Dang L, Ganz T, Jung CL, Iolascon A, Brugnara C, De Franceschi L. Matte A, et al. Among authors: kosinski pa. J Clin Invest. 2021 May 17;131(10):e144206. doi: 10.1172/JCI144206. J Clin Invest. 2021. PMID: 33822774 Free PMC article.
Phase 1 Single- and Multiple-Ascending-Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers.
Yang H, Merica E, Chen Y, Cohen M, Goldwater R, Kosinski PA, Kung C, Yuan ZJ, Silverman L, Goldwasser M, Silver BA, Agresta S, Barbier AJ. Yang H, et al. Among authors: kosinski pa. Clin Pharmacol Drug Dev. 2019 Feb;8(2):246-259. doi: 10.1002/cpdd.604. Epub 2018 Aug 9. Clin Pharmacol Drug Dev. 2019. PMID: 30091852 Clinical Trial.
The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency.
Al-Samkari H, Addonizio K, Glader B, Morton DH, Chonat S, Thompson AA, Kuo KHM, Ravindranath Y, Wang H, Rothman JA, Kwiatkowski JL, Kung C, Kosinski PA, Al-Sayegh H, London WB, Grace RF. Al-Samkari H, et al. Among authors: kosinski pa. Br J Haematol. 2021 Mar;192(6):1092-1096. doi: 10.1111/bjh.16724. Epub 2020 May 28. Br J Haematol. 2021. PMID: 32463523 Free article. Clinical Trial.
Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
Garcia-Gomez M, Calabria A, Garcia-Bravo M, Benedicenti F, Kosinski P, López-Manzaneda S, Hill C, Del Mar Mañu-Pereira M, Martín MA, Orman I, Vives-Corrons JL, Kung C, Schambach A, Jin S, Bueren JA, Montini E, Navarro S, Segovia JC. Garcia-Gomez M, et al. Mol Ther. 2016 Aug;24(7):1187-98. doi: 10.1038/mt.2016.87. Epub 2016 May 3. Mol Ther. 2016. PMID: 27138040 Free PMC article.
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