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81 results

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Page 1
Optimizing liver health before and after gene therapy for hemophilia A.
Ragni MV, Mead H, de Jong YP, Kaczmarek R, Leavitt AD, Long B, Nugent DJ, Sabatino DE, Fong S, von Drygalski A, Walsh CE, Luxon BA. Ragni MV, et al. Among authors: leavitt ad. Blood Adv. 2024 Oct 8;8(19):5203-5212. doi: 10.1182/bloodadvances.2024013059. Blood Adv. 2024. PMID: 38843379 Free PMC article. Review.
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: leavitt ad. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a phase 3, open-label, randomised, crossover trial.
Ragni MV, Rothenberger SD, Feldman R, Nance D, Leavitt AD, Malec L, Kulkarni R, Sidonio R Jr, Kraut E, Lasky J, Pruthi R, Angelini D, Philipp C, Hwang N, Wheeler AP, Seaman C, Machin N, Xavier F, Meyer M, Bellissimo D, Humphreys G, Smith KJ, Merricks EP, Nichols TC, Ivanco D, Vehec D, Koerbel G, Althouse AD. Ragni MV, et al. Among authors: leavitt ad. Lancet Haematol. 2023 Aug;10(8):e612-e623. doi: 10.1016/S2352-3026(23)00119-9. Epub 2023 Jun 26. Lancet Haematol. 2023. PMID: 37385272 Free PMC article. Clinical Trial.
Observational cohort study of long-term outcomes of liver transplantation in haemophilia.
Ragni MV, Callis J, Daoud N, Hu B, Manuel M, Santos J, Schwartz J, Friedman KD, Kouides P, Kuriakose P, Leavitt AD, Lim MY, Machin N, Recht M, Chrisentery-Singleton T. Ragni MV, et al. Among authors: leavitt ad. Haemophilia. 2024 Jan;30(1):87-97. doi: 10.1111/hae.14910. Epub 2023 Dec 18. Haemophilia. 2024. PMID: 38111071
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A.
Madan B, Ozelo MC, Raheja P, Symington E, Quon DV, Leavitt AD, Pipe SW, Lowe G, Kenet G, Reding MT, Mason J, Wang M, von Drygalski A, Klamroth R, Shapiro S, Chambost H, Dunn AL, Oldenburg J, Chou SC, Peyvandi F, Millar CM, Osmond D, Yu H, Dashiell-Aje E, Robinson TM, Mahlangu J. Madan B, et al. Among authors: leavitt ad. J Thromb Haemost. 2024 Jul;22(7):1880-1893. doi: 10.1016/j.jtha.2024.04.001. Epub 2024 Apr 12. J Thromb Haemost. 2024. PMID: 38614387 Free article. Clinical Trial.
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.
Cuker A, Kavakli K, Frenzel L, Wang JD, Astermark J, Cerqueira MH, Iorio A, Katsarou-Fasouli O, Klamroth R, Shapiro AD, Hermans C, Ishiguro A, Leavitt AD, Oldenburg JB, Ozelo MC, Teitel J, Biondo F, Fang A, Fuiman J, McKay J, Sun P, Rasko JEJ, Rupon J; BENEGENE-2 Trial Investigators. Cuker A, et al. Among authors: leavitt ad. N Engl J Med. 2024 Sep 26;391(12):1108-1118. doi: 10.1056/NEJMoa2302982. N Engl J Med. 2024. PMID: 39321362 Clinical Trial.
Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial.
Leavitt AD, Mahlangu J, Raheja P, Symington E, Quon DV, Giermasz A, López Fernández MF, Kenet G, Lowe G, Key NS, Millar CM, Pipe SW, Madan B, Chou SC, Klamroth R, Mason J, Chambost H, Peyvandi F, Majerus E, Pepperell D, Rivat C, Yu H, Robinson TM, Ozelo MC. Leavitt AD, et al. Res Pract Thromb Haemost. 2024 Oct 30;8(8):102615. doi: 10.1016/j.rpth.2024.102615. eCollection 2024 Nov. Res Pract Thromb Haemost. 2024. PMID: 39687929 Free PMC article.
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1.
O'Mahony B, Dunn AL, Leavitt AD, Peyvandi F, Ozelo MC, Mahlangu J, Peerlinck K, Wang JD, Lowe GC, Tan CW, Giermasz A, Tran H, Khoo TL, Cockrell E, Pepperell D, Chambost H, López Fernández MF, Kazmi R, Majerus E, Skinner MW, Klamroth R, Quinn J, Yu H, Wong WY, Robinson TM, Pipe SW. O'Mahony B, et al. Among authors: leavitt ad. J Thromb Haemost. 2023 Dec;21(12):3450-3462. doi: 10.1016/j.jtha.2023.08.032. Epub 2023 Sep 6. J Thromb Haemost. 2023. PMID: 37678546 Free article. Clinical Trial.
81 results