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524 results

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Page 1
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: rangarajan s. N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10. N Engl J Med. 2011. PMID: 22149959 Free PMC article. Clinical Trial.
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: rangarajan s. N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309. N Engl J Med. 2014. PMID: 25409372 Free PMC article. Clinical Trial.
Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.
Pasi KJ, Rangarajan S, Georgiev P, Mant T, Creagh MD, Lissitchkov T, Bevan D, Austin S, Hay CR, Hegemann I, Kazmi R, Chowdary P, Gercheva-Kyuchukova L, Mamonov V, Timofeeva M, Soh CH, Garg P, Vaishnaw A, Akinc A, Sørensen B, Ragni MV. Pasi KJ, et al. Among authors: rangarajan s. N Engl J Med. 2017 Aug 31;377(9):819-828. doi: 10.1056/NEJMoa1616569. Epub 2017 Jul 10. N Engl J Med. 2017. PMID: 28691885 Free article. Clinical Trial.
AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.
Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, Yu H, Vettermann C, Pierce GF, Wong WY, Pasi KJ. Rangarajan S, et al. N Engl J Med. 2017 Dec 28;377(26):2519-2530. doi: 10.1056/NEJMoa1708483. Epub 2017 Dec 9. N Engl J Med. 2017. PMID: 29224506 Free article. Clinical Trial.
Adenovirus-associated antibodies in UK cohort of hemophilia patients: A seroprevalence study of the presence of adenovirus-associated virus vector-serotypes AAV5 and AAV8 neutralizing activity and antibodies in patients with hemophilia A.
Stanford S, Pink R, Creagh D, Clark A, Lowe G, Curry N, Pasi J, Perry D, Fong S, Hayes G, Chandrakumaran K, Rangarajan S. Stanford S, et al. Among authors: rangarajan s. Res Pract Thromb Haemost. 2019 Jan 25;3(2):261-267. doi: 10.1002/rth2.12177. eCollection 2019 Apr. Res Pract Thromb Haemost. 2019. PMID: 31011710 Free PMC article.
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.
Pasi KJ, Rangarajan S, Mitchell N, Lester W, Symington E, Madan B, Laffan M, Russell CB, Li M, Pierce GF, Wong WY. Pasi KJ, et al. Among authors: rangarajan s. N Engl J Med. 2020 Jan 2;382(1):29-40. doi: 10.1056/NEJMoa1908490. N Engl J Med. 2020. PMID: 31893514 Clinical Trial.
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. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
524 results