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Page 1
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. 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. Among authors: ozelo mc. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
Cost of hemophilia A in Brazil: a microcosting study.
da Silva Etges APB, Schneider NB, Roos EC, Marcolino MAZ, Ozelo MC, Midori Takahashi Hosokawa Nikkuni M, Elvira Mesquita Carvalho L, Oliveira Rebouças T, Hermida Cerqueira M, Mata V, Polanczyk CA. da Silva Etges APB, et al. Among authors: ozelo mc. Health Econ Rev. 2024 Aug 6;14(1):62. doi: 10.1186/s13561-024-00539-x. Health Econ Rev. 2024. PMID: 39105856 Free PMC article.
Safety and efficacy of long-term emicizumab prophylaxis in hemophilia A with factor VIII inhibitors: A phase 3b, multicenter, single-arm study (STASEY).
Jiménez-Yuste V, Peyvandi F, Klamroth R, Castaman G, Shanmukhaiah C, Rangarajan S, García Chavez J, Martinez R, Kenet G, Alzahrani H, Robson S, Schmitt C, Kiialainen A, Meier O, Ozelo M. Jiménez-Yuste V, et al. Res Pract Thromb Haemost. 2022 Nov 14;6(8):e12837. doi: 10.1002/rth2.12837. eCollection 2022 Nov. Res Pract Thromb Haemost. 2022. PMID: 36397934 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: ozelo mc. 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.
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