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382 results

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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.
Predictive performance of pharmacokinetic-guided prophylactic dosing of factor concentrates in hemophilia A and B.
Goedhart TMHJ, Bukkems LH, Zwagemaker AF, Coppens M, Fijnvandraat K, Schols SEM, Schutgens REG, Eikenboom J, Heubel-Moenen FCJI, Ypma PF, Nieuwenhuizen L, Meijer K, Leebeek FWG, Mathôt RAA, Cnossen MH. Goedhart TMHJ, et al. Among authors: leebeek fwg. Res Pract Thromb Haemost. 2024 Mar 28;8(3):102397. doi: 10.1016/j.rpth.2024.102397. eCollection 2024 Mar. Res Pract Thromb Haemost. 2024. PMID: 38689619 Free PMC article.
Variant mapping using mass spectrometry-based proteotyping as a diagnostic tool in Von Willebrand Disease.
Kreft IC, van Duijl TT, van Kwawegen C, Atiq F, Phan W, Schuller MBP, Boon-Spijker M, van der Zwaan C, Meijer AB, Hoogendijk AJ, Bierings R, Eikenboom JCJ, Leebeek FWG, van den Biggelaar M; WiN study group. Kreft IC, et al. Among authors: leebeek fwg. J Thromb Haemost. 2024 Apr 26:S1538-7836(24)00231-9. doi: 10.1016/j.jtha.2024.04.011. Online ahead of print. J Thromb Haemost. 2024. PMID: 38679335 Free article.
Tachyphylaxis and reproducibility of desmopressin response in perioperative persons with nonsevere hemophilia A: implications for clinical practice.
Romano LGR, Schütte LM, van Hest RM, Meijer K, Laros-van Gorkom BAP, Nieuwenhuizen L, Eikenboom J, Heubel-Moenen FCJI, Uitslager N, Coppens M, Fijnvandraat K, Driessens MHE, Polinder S, Cnossen MH, Leebeek FWG, Mathôt RAA, Kruip MJHA; DAVID and SYMPHONY Consortium. Romano LGR, et al. Among authors: leebeek fwg. Res Pract Thromb Haemost. 2024 Mar 5;8(3):102367. doi: 10.1016/j.rpth.2024.102367. eCollection 2024 Mar. Res Pract Thromb Haemost. 2024. PMID: 38660455 Free PMC article.
382 results