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Page 1
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Pipe SW, et al. Among authors: leebeek fwg. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy.
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key NS, Lattimore SU, Leebeek FWG, Miesbach WA, Recht M, Gut R, Dolmetsch R, Monahan PE, Le Quellec S, Pipe SW. von Drygalski A, et al. Among authors: leebeek fwg. Blood Adv. 2023 Oct 10;7(19):5671-5679. doi: 10.1182/bloodadvances.2022008886. Blood Adv. 2023. PMID: 36490302 Free PMC article.
Antithrombotic Treatment in Patients With Hemophilia: an EHA-ISTH-EAHAD-ESO Clinical Practice Guidance.
Schutgens REG, Jimenez-Yuste V, Escobar M, Falanga A, Gigante B, Klamroth R, Lassila R, Leebeek FWG, Makris M, Owaidah T, Sholzberg M, Tiede A, Werring DJ, van der Worp HB, Windyga J, Castaman G. Schutgens REG, et al. Among authors: leebeek fwg. Hemasphere. 2023 Jun 5;7(6):e900. doi: 10.1097/HS9.0000000000000900. eCollection 2023 Jun. Hemasphere. 2023. PMID: 37304933 Free PMC article.
A new population pharmacokinetic model for recombinant factor IX-Fc fusion concentrate including young children with haemophilia B.
Koopman SF, Goedhart TMHJ, Bukkems LH, Mulders TM, Leebeek FWG, Fijnvandraat K, Coppens M, Mathias M, Collins PW, Tait RC, Bagot CN, Curry N, Payne J, Chowdary P, Cnossen MH, Mathôt RAA; OPTI-CLOT study group and SYMPHONY consortium. Koopman SF, et al. Among authors: leebeek fwg. Br J Clin Pharmacol. 2024 Jan;90(1):220-231. doi: 10.1111/bcp.15881. Epub 2023 Sep 12. Br J Clin Pharmacol. 2024. PMID: 37567779
Perioperative pharmacokinetic-guided factor VIII concentrate dosing in haemophilia (OPTI-CLOT trial): an open-label, multicentre, randomised, controlled trial.
van Moort I, Preijers T, Bukkems LH, Hazendonk HCAM, van der Bom JG, Laros-van Gorkom BAP, Beckers EAM, Nieuwenhuizen L, van der Meer FJM, Ypma P, Coppens M, Fijnvandraat K, Schutgens REG, Meijer K, Leebeek FWG, Mathôt RAA, Cnossen MH; OPTI-CLOT study group. van Moort I, et al. Among authors: leebeek fwg. Lancet Haematol. 2021 Jul;8(7):e492-e502. doi: 10.1016/S2352-3026(21)00135-6. Lancet Haematol. 2021. PMID: 34171280 Clinical Trial.
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.
391 results