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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.
Phase 3 Trial of Concizumab in Hemophilia with Inhibitors.
Matsushita T, Shapiro A, Abraham A, Angchaisuksiri P, Castaman G, Cepo K, d'Oiron R, Frei-Jones M, Goh AS, Haaning J, Hald Jacobsen S, Mahlangu J, Mathias M, Nogami K, Skovgaard Rasmussen J, Stasyshyn O, Tran H, Vilchevska K, Villarreal Martinez L, Windyga J, You CW, Zozulya N, Zulfikar B, Jiménez-Yuste V; explorer7 Investigators. Matsushita T, et al. N Engl J Med. 2023 Aug 31;389(9):783-794. doi: 10.1056/NEJMoa2216455. N Engl J Med. 2023. PMID: 37646676 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.
ADAMTS13 and TTP.
Zheng X, Majerus EM, Sadler JE. Zheng X, et al. Among authors: majerus em. Curr Opin Hematol. 2002 Sep;9(5):389-94. doi: 10.1097/00062752-200209000-00001. Curr Opin Hematol. 2002. PMID: 12172456 Review.
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: majerus e. 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 Clinical Trial.
Valoctocogene roxaparvovec gene transfer in participants with HIV.
Ragni MV, Majerus E, Fong S, Yates B, Scheeler S, Razon L, Yu H, Reddy DB, Robinson TM. Ragni MV, et al. Among authors: majerus e. Blood Adv. 2023 Apr 25;7(8):1525-1530. doi: 10.1182/bloodadvances.2022008948. Blood Adv. 2023. PMID: 36696462 Free PMC article. No abstract available.
O-fucosylation is required for ADAMTS13 secretion.
Ricketts LM, Dlugosz M, Luther KB, Haltiwanger RS, Majerus EM. Ricketts LM, et al. Among authors: majerus em. J Biol Chem. 2007 Jun 8;282(23):17014-23. doi: 10.1074/jbc.M700317200. Epub 2007 Mar 29. J Biol Chem. 2007. PMID: 17395589 Free article.
Binding of ADAMTS13 to von Willebrand factor.
Majerus EM, Anderson PJ, Sadler JE. Majerus EM, et al. J Biol Chem. 2005 Jun 10;280(23):21773-8. doi: 10.1074/jbc.M502529200. Epub 2005 Apr 11. J Biol Chem. 2005. PMID: 15824096 Free article.
24 results