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88 results

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Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study.
Kenet G, Chen YC, Lowe G, Percy C, Tran H, von Drygalski A, Trossaërt M, Reding M, Oldenburg J, Mingot-Castellano ME, Park YS, Peyvandi F, Ozelo MC, Mahlangu J, Quinn J, Huang M, Reddy DB, Kim B. Kenet G, et al. Among authors: von drygalski a. J Clin Med. 2021 Dec 18;10(24):5959. doi: 10.3390/jcm10245959. J Clin Med. 2021. PMID: 34945255 Free PMC article.
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A.
Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators. Mahlangu J, et al. Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13. Blood. 2014. PMID: 24227821 Free PMC article. 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.
Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K; XTEND-1 Trial Group. von Drygalski A, et al. N Engl J Med. 2023 Jan 26;388(4):310-318. doi: 10.1056/NEJMoa2209226. N Engl J Med. 2023. PMID: 36720133 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: von drygalski a. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
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: von drygalski a. J Thromb Haemost. 2024 Apr 12:S1538-7836(24)00184-3. doi: 10.1016/j.jtha.2024.04.001. Online ahead of print. J Thromb Haemost. 2024. PMID: 38614387 Free article.
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.
Tran DQ, Benson CC, Boice JA, Chitlur M, Dunn AL, Escobar MA, Gupta K, Johnsen JM, Jorgenson J, Martin SD, Martin S, Meeks SL, Narvaez AA Jr, Quon DV, Reding MT, Reiss UM, Savage B, Schafer K, Steiner B, Thornburg C, Volland LM, von Drygalski A. Tran DQ, et al. Among authors: von drygalski a. Expert Rev Hematol. 2023 Mar;16(sup1):19-37. doi: 10.1080/17474086.2023.2171981. Expert Rev Hematol. 2023. PMID: 36920859 Free PMC article.
88 results