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Page 1
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
Mahlangu J, Kaczmarek R, von Drygalski A, Shapiro S, Chou SC, Ozelo MC, Kenet G, Peyvandi F, Wang M, Madan B, Key NS, Laffan M, Dunn AL, Mason J, Quon DV, Symington E, Leavitt AD, Oldenburg J, Chambost H, Reding MT, Jayaram K, Yu H, Mahajan R, Chavele KM, Reddy DB, Henshaw J, Robinson TM, Wong WY, Pipe SW; GENEr8-1 Trial Group. Mahlangu J, et al. Among authors: pipe sw. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
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. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial.
Srivastava A, Rangarajan S, Kavakli K, Klamroth R, Kenet G, Khoo L, You CW, Xu W, Malan N, Frenzel L, Bagot CN, Stasyshyn O, Chang CY, Poloskey S, Qiu Z, Andersson S, Mei B, Pipe SW. Srivastava A, et al. Among authors: pipe sw. Lancet Haematol. 2023 May;10(5):e322-e332. doi: 10.1016/S2352-3026(23)00037-6. Epub 2023 Mar 29. Lancet Haematol. 2023. PMID: 37003278 Clinical Trial.
Corrigendum to 'Efficacy of emicizumab is maintained throughout dosing intervals for bleed prophylaxis' [Research and Practice in Thrombosis and Haemostasis, Volume 7, Issue 2, February 2023, 100077].
Pipe SW, Trzaskoma B, Minhas M, Lehle M, Ko RH, Gao L, Mahlangu J, Kempton CL, Kessler CM, Kruse-Jarres R. Pipe SW, et al. Res Pract Thromb Haemost. 2023 May 27;7(4):100191. doi: 10.1016/j.rpth.2023.100191. eCollection 2023 May. Res Pract Thromb Haemost. 2023. PMID: 37538504 Free PMC article.
Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy.
Miesbach W, Konkle B, Chowdary P, Kaczmarek R, Leebeek F, Mahlangu J, Makris M, Pipe SW, Srivastava A, Voorberg J, Pierce GF, Peyvandi F. Miesbach W, et al. Among authors: pipe sw. J Thromb Haemost. 2024 May;22(5):1510-1515. doi: 10.1016/j.jtha.2023.12.039. Epub 2024 Jan 18. J Thromb Haemost. 2024. PMID: 38242208
Pharmacokinetics and coagulation biomarkers in children and adults with hemophilia A receiving emicizumab prophylaxis every 1, 2, or 4 weeks.
Kiialainen A, Adamkewicz JI, Petry C, Oldenburg J, Pipe SW, Young G, Mahlangu J, Lehle M, Niggli M, Castaman G, Jiménez-Yuste V, Shima M, Négrier C, Schmitt C. Kiialainen A, et al. Among authors: pipe sw. Res Pract Thromb Haemost. 2023 Dec 29;8(1):102306. doi: 10.1016/j.rpth.2023.102306. eCollection 2024 Jan. Res Pract Thromb Haemost. 2023. PMID: 38282901 Free PMC article.
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
Coppens M, Pipe SW, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan PE, Leebeek FWG; HOPE-B Investigators. Coppens M, et al. Among authors: pipe sw. Lancet Haematol. 2024 Apr;11(4):e265-e275. doi: 10.1016/S2352-3026(24)00006-1. Epub 2024 Mar 1. Lancet Haematol. 2024. PMID: 38437857 Clinical Trial.
148 results