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111 results

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Page 1
Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort.
Pasi KJ, Lissitchkov T, Mamonov V, Mant T, Timofeeva M, Bagot C, Chowdary P, Georgiev P, Gercheva-Kyuchukova L, Madigan K, Van Nguyen H, Yu Q, Mei B, Benson CC, Ragni MV. Pasi KJ, et al. J Thromb Haemost. 2021 Jun;19(6):1436-1446. doi: 10.1111/jth.15270. Epub 2021 May 18. J Thromb Haemost. 2021. PMID: 33587824 Free PMC article. Clinical Trial.
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. Among authors: pasi kj. 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.
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B.
Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators. Powell JS, et al. Among authors: pasi kj. N Engl J Med. 2013 Dec 12;369(24):2313-23. doi: 10.1056/NEJMoa1305074. Epub 2013 Dec 4. N Engl J Med. 2013. PMID: 24304002 Free article. Clinical Trial.
Long-term safety and efficacy of extended-interval prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) in subjects with haemophilia B.
Pasi KJ, Fischer K, Ragni M, Nolan B, Perry DJ, Kulkarni R, Ozelo M, Mahlangu J, Shapiro AD, Baker RI, Bennett CM, Barnes C, Oldenburg J, Matsushita T, Yuan H, Ramirez-Santiago A, Pierce GF, Allen G, Mei B. Pasi KJ, et al. Thromb Haemost. 2017 Feb 28;117(3):508-518. doi: 10.1160/TH16-05-0398. Epub 2016 Dec 22. Thromb Haemost. 2017. PMID: 28004057 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. 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.
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. Haemophilia. 2020 Nov;26(6):e262-e271. doi: 10.1111/hae.14036. Epub 2020 Jun 4. Haemophilia. 2020. PMID: 32497409
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. Among authors: pasi kj. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies.
Chowdary P, Holmström M, Mahlangu JN, Ozelo MC, Pabinger I, Pasi KJ, Ragni MV, Shapiro A, Barnowski C, Lethagen S. Chowdary P, et al. Among authors: pasi kj. Res Pract Thromb Haemost. 2022 Jul 26;6(5):e12760. doi: 10.1002/rth2.12760. eCollection 2022 Jul. Res Pract Thromb Haemost. 2022. PMID: 35910942 Free PMC article.
Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B.
Shapiro AD, Kulkarni R, Ragni MV, Chambost H, Mahlangu J, Oldenburg J, Nolan B, Ozelo MC, Foster MC, Willemze A, Barnowski C, Jain N, Winding B, Dumont J, Lethagen S, Barnes C, Pasi KJ. Shapiro AD, et al. Among authors: pasi kj. Blood Adv. 2023 Jul 11;7(13):3049-3057. doi: 10.1182/bloodadvances.2022009230. Blood Adv. 2023. PMID: 36848635 Free PMC article.
111 results