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53 results

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Page 1
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. Among authors: giermasz a. 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.
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Ozelo MC, et al. Among authors: giermasz a. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
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. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
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: giermasz a. 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.
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: giermasz a. Blood Adv. 2023 Oct 10;7(19):5671-5679. doi: 10.1182/bloodadvances.2022008886. Blood Adv. 2023. PMID: 36490302 Free PMC article.
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study.
Leavitt AD, Konkle BA, Stine KC, Visweshwar N, Harrington TJ, Giermasz A, Arkin S, Fang A, Plonski F, Yver A, Ganne F, Agathon D, Resa MLA, Tseng LJ, Di Russo G, Cockroft BM, Cao L, Rupon J. Leavitt AD, et al. Among authors: giermasz a. Blood. 2024 Feb 29;143(9):796-806. doi: 10.1182/blood.2022018971. Blood. 2024. PMID: 37871576 Free PMC article. Clinical Trial.
Bleed treatment with eptacog beta (rFVIIa) results in a low incidence of rebleeding in adult and adolescent patients with haemophilia A or B with inhibitors.
Dunn A, Dargaud Y, Abajas Y, Carcao M, Castaman G, Giermasz A, Hermans C, Jiménez-Yuste V, Lewandowska M, Mahlangu J, Meeks S, Miesbach W, Recht M, Salinas V, Chrisentery-Singleton T, Bonzo D, Mitchell IS, Wilkinson TA, Young G. Dunn A, et al. Among authors: giermasz a. Haemophilia. 2024 Dec 15. doi: 10.1111/hae.15109. Online ahead of print. Haemophilia. 2024. PMID: 39676340
PERSEPT 3: A phase 3 clinical trial to evaluate the haemostatic efficacy of eptacog beta (recombinant human FVIIa) in perioperative care in subjects with haemophilia A or B with inhibitors.
Escobar M, Luck J, Averianov Y, Ducore J, Fernández MFL, Giermasz A, Hart DP, Journeycake J, Kessler C, Leissinger C, Mahlangu J, Martinez LV, Miesbach W, Mitha IH, Quon D, Reding MT, Schved JF, Stasyshyn O, Vilchevska KV, Wang M, Windyga J, Alexander WA, Al-Sabbagh A, Bonzo D, Mitchell IS, Wilkinson TA, Hermans C. Escobar M, et al. Among authors: giermasz a. Haemophilia. 2021 Nov;27(6):911-920. doi: 10.1111/hae.14418. Epub 2021 Oct 6. Haemophilia. 2021. PMID: 34614267 Free PMC article. Clinical Trial.
53 results