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119 results

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Page 1
Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A.
Klamroth R, Hayes G, Andreeva T, Gregg K, Suzuki T, Mitha IH, Hardesty B, Shima M, Pollock T, Slev P, Oldenburg J, Ozelo MC, Stieltjes N, Castet SM, Mahlangu J, Peyvandi F, Kazmi R, Schved JF, Leavitt AD, Callaghan M, Pan-Petesch B, Quon DV, Andrews J, Trinh A, Li M, Wong WY. Klamroth R, et al. Among authors: ozelo mc. Hum Gene Ther. 2022 Apr;33(7-8):432-441. doi: 10.1089/hum.2021.287. Epub 2022 Mar 16. Hum Gene Ther. 2022. PMID: 35156839 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.
Key issues in inhibitor management in patients with haemophilia.
Gomez K, Klamroth R, Mahlangu J, Mancuso ME, Mingot ME, Ozelo MC. Gomez K, et al. Among authors: ozelo mc. Blood Transfus. 2014 Jan;12 Suppl 1(Suppl 1):s319-29. doi: 10.2450/2013.0246-12. Epub 2013 Dec 3. Blood Transfus. 2014. PMID: 24333092 Free PMC article. Review. No abstract available.
Interim results from a large multinational extension trial (guardian(™) 2) using turoctocog alfa for prophylaxis and treatment of bleeding in patients with severe haemophilia A.
Lentz SR, Cerqueira M, Janic D, Kempton C, Matytsina I, Misgav M, Oldenburg J, Ozelo M, Recht M, Rosholm A, Savic A, Suzuki T, Tiede A, Santagostino E. Lentz SR, et al. Haemophilia. 2016 Sep;22(5):e445-9. doi: 10.1111/hae.12990. Epub 2016 Jun 13. Haemophilia. 2016. PMID: 27291066 Clinical Trial. No abstract available.
Long-term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B-YOND extension study.
Pasi KJ, Fischer K, Ragni M, Kulkarni R, Ozelo MC, Mahlangu J, Shapiro A, P'Ng S, Chambost H, Nolan B, Bennett C, Matsushita T, Winding B, Fruebis J, Yuan H, Rudin D, Oldenburg J. Pasi KJ, et al. Among authors: ozelo mc. Haemophilia. 2020 Nov;26(6):e262-e271. doi: 10.1111/hae.14036. Epub 2020 Jun 4. Haemophilia. 2020. PMID: 32497409
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BJ, Ozelo M, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA. George LA, et al. Mol Ther. 2020 Sep 2;28(9):2073-2082. doi: 10.1016/j.ymthe.2020.06.001. Epub 2020 Jun 10. Mol Ther. 2020. PMID: 32559433 Free PMC article. Clinical Trial.
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: ozelo mc. J Clin Med. 2021 Dec 18;10(24):5959. doi: 10.3390/jcm10245959. J Clin Med. 2021. PMID: 34945255 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. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
119 results