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Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs.
Linthorst NA, Jongejan YK, Dirven RJ, Laan SNJ, Bierings R, Casari C, Cordfunke RA, Dahlman JE, Dolezal N, Drijfhout JW, Leebeek FWG, Ruhaak LR, Schrader Echeverri E, Voorberg J, van Vlijmen BJM, Denis CV, Eikenboom JCJ. Linthorst NA, et al. Among authors: leebeek fwg. Blood Adv. 2025 Jan 28;9(2):310-320. doi: 10.1182/bloodadvances.2024014601. Blood Adv. 2025. PMID: 39820471
Prediction of the chance of successful immune tolerance induction in persons with severe hemophilia A and inhibitors: a clinical prediction model.
Oomen I, Abdi A, Camelo RM, Callado FMRA, Carvalho LEM, Calcaterra IL, Carcao M, Castaman G, Eikenboom JCJ, Fischer K, Franco VKB, Heymans MW, Leebeek FWG, Lillicrap D, Lorenzato CS, Mancuso ME, Matino D, Di Minno MND, Mohseny AB, Oldenburg J, Rezende SM, Rivard GE, Rydz N, Schols SEM, Voorberg J, Fijnvandraat K, Gouw SC; International Genetic and clinical determinants for the outcome of immune tolerance induction study group. Oomen I, et al. Among authors: leebeek fwg. Res Pract Thromb Haemost. 2024 Oct 3;8(7):102580. doi: 10.1016/j.rpth.2024.102580. eCollection 2024 Oct. Res Pract Thromb Haemost. 2024. PMID: 39558913 Free PMC article.
The severe von Willebrand disease variant p.M771V leads to impaired anterograde trafficking of von Willebrand factor in patient-derived and base-edited endothelial colony-forming cells.
Bär I, Barraclough A, Bürgisser PE, van Kwawegen C, Fijnvandraat K, Eikenboom JCJ, Leebeek FWG, Voorberg J, Bierings R. Bär I, et al. Among authors: leebeek fwg. J Thromb Haemost. 2024 Nov 6:S1538-7836(24)00639-1. doi: 10.1016/j.jtha.2024.10.023. Online ahead of print. J Thromb Haemost. 2024. PMID: 39510415 Free article.
Patient-reported data on the severity of Von Willebrand disease.
van Kwawegen CB, Fijnvandraat K, Kruip MJHA, de Meris J, Schols SEM, Meijer K, van der Bom JG, Cnossen MH, van Galen KPM, Atiq F, Eikenboom J, Leebeek FWG; WiN study group. van Kwawegen CB, et al. Among authors: leebeek fwg. Haemophilia. 2024 Nov;30(6):1348-1356. doi: 10.1111/hae.15103. Epub 2024 Oct 15. Haemophilia. 2024. PMID: 39403864 Free PMC article.
Genetic variants, thrombocytopenia, and clinical phenotype of type 2B von Willebrand disease: a median 16-year follow-up study.
van Kwawegen CB, Atiq F, Endenburg D, Fijnvandraat K, van Galen KPM, Cnossen MH, Schols SEM, Kruip MJHA, van Heerde WL, de Meris J, van der Bom JG, Eikenboom J, Meijer K, Leebeek FWG; WiN study group. van Kwawegen CB, et al. Among authors: leebeek fwg. J Thromb Haemost. 2024 Dec;22(12):3460-3472. doi: 10.1016/j.jtha.2024.08.028. Epub 2024 Sep 28. J Thromb Haemost. 2024. PMID: 39343102 Free article.
Psychometrics of patient-reported outcomes measurement information system in von Willebrand disease, inherited platelet function disorders, and rare bleeding disorders.
van Hoorn ES, Willems SPE, Al Arashi W, de Moor AS, van Kwawegen CB, Teela L, Oude Voshaar MAH, Kremer Hovinga ICL, Schutgens REG, Schols SEM, Leebeek FWG, Haverman L, Cnossen MH, Gouw SC, Lingsma HF; SYMPHONY consortium and the Dutch research group for PROMIS implementation in inherited bleeding disorders. van Hoorn ES, et al. Among authors: leebeek fwg. Res Pract Thromb Haemost. 2024 Jun 13;8(4):102474. doi: 10.1016/j.rpth.2024.102474. eCollection 2024 May. Res Pract Thromb Haemost. 2024. PMID: 39076727 Free PMC article.
436 results