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Items: 7

1.

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.

Haematologica. 2020 Jan 23. pii: haematol.2019.238865. doi: 10.3324/haematol.2019.238865. [Epub ahead of print]

2.

Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study.

van Beers EJ, van Straaten S, Morton DH, Barcellini W, Eber SW, Glader B, Yaish HM, Chonat S, Kwiatkowski JL, Rothman JA, Sharma M, Neufeld EJ, Sheth S, Despotovic JM, Kollmar N, Pospíšilová D, Knoll CM, Kuo K, Pastore YD, Thompson AA, Newburger PE, Ravindranath Y, Wang WC, Wlodarski MW, Wang H, Holzhauer S, Breakey VR, Verhovsek M, Kunz J, McNaull MA, Rose MJ, Bradeen HA, Addonizio K, Li A, Al-Sayegh H, London WB, Grace RF.

Haematologica. 2019 Feb;104(2):e51-e53. doi: 10.3324/haematol.2018.196295. Epub 2018 Sep 13. No abstract available.

3.

Organ involvement occurs in all forms of hereditary haemolytic anaemia.

van Straaten S, Verhoeven J, Hagens S, Schutgens R, van Solinge W, van Wijk R, van Beers EJ.

Br J Haematol. 2019 May;185(3):602-605. doi: 10.1111/bjh.15575. Epub 2018 Sep 5. No abstract available.

PMID:
30183081
4.

Iron overload in patients with rare hereditary hemolytic anemia: Evidence-based suggestion on whom and how to screen.

van Straaten S, Biemond BJ, Kerkhoffs JL, Gitz-Francois J, van Wijk R, van Beers EJ.

Am J Hematol. 2018 Nov;93(11):E374-E376. doi: 10.1002/ajh.25251. Epub 2018 Sep 21. No abstract available.

5.

Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency.

van Straaten S, Bierings M, Bianchi P, Akiyoshi K, Kanno H, Serra IB, Chen J, Huang X, van Beers E, Ekwattanakit S, Güngör T, Kors WA, Smiers F, Raymakers R, Yanez L, Sevilla J, van Solinge W, Segovia JC, van Wijk R.

Haematologica. 2018 Feb;103(2):e82-e86. doi: 10.3324/haematol.2017.177857. Epub 2017 Dec 14. No abstract available.

6.

Treatment of vitamin D insufficiency in children and adolescents with inflammatory bowel disease: a randomized clinical trial comparing three regimens.

Pappa HM, Mitchell PD, Jiang H, Kassiff S, Filip-Dhima R, DiFabio D, Quinn N, Lawton RC, Varvaris M, Van Straaten S, Gordon CM.

J Clin Endocrinol Metab. 2012 Jun;97(6):2134-42. doi: 10.1210/jc.2011-3182. Epub 2012 Mar 28.

7.

Distinctive roles of neutrophils and monocytes in anti-thy-1 nephritis.

Westerhuis R, van Straaten SC, van Dixhoorn MG, van Rooijen N, Verhagen NA, Dijkstra CD, de Heer E, Daha MR.

Am J Pathol. 2000 Jan;156(1):303-10.

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