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443 results

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Page 1
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Manno CS, et al. Among authors: ragni m. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 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: ragni mv. 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.
Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates.
Powell J, Shapiro A, Ragni M, Negrier C, Windyga J, Ozelo M, Pasi J, Baker R, Potts J, Li S, Mei B, Pierce GF, Robinson B. Powell J, et al. Among authors: ragni m. Br J Haematol. 2015 Jan;168(1):113-23. doi: 10.1111/bjh.13109. Epub 2014 Sep 11. Br J Haematol. 2015. PMID: 25209873 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. Among authors: ragni m. 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.
Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults.
Konkle BA, Oldenburg J, Pasi J, Kulkarni R, Nolan B, Mahlangu J, Young G, Brown SA, Pabinger I, Shapiro A, Négrier C, Blanchette V, Ragni MV, Dumont J, Lethagen S. Konkle BA, et al. Among authors: ragni mv. Res Pract Thromb Haemost. 2023 Aug 22;7(6):102180. doi: 10.1016/j.rpth.2023.102180. eCollection 2023 Aug. Res Pract Thromb Haemost. 2023. PMID: 37753224 Free PMC article.
Clinical evaluation of recombinant factor IX.
White G, Shapiro A, Ragni M, Garzone P, Goodfellow J, Tubridy K, Courter S. White G, et al. Among authors: ragni m. Semin Hematol. 1998 Apr;35(2 Suppl 2):33-8. Semin Hematol. 1998. PMID: 9565165 Review.
A cross-sectional analysis of cardiovascular disease in the hemophilia population.
Sood SL, Cheng D, Ragni M, Kessler CM, Quon D, Shapiro AD, Key NS, Manco-Johnson MJ, Cuker A, Kempton C, Wang TF, Eyster ME, Kuriakose P, von Drygalski A, Gill JC, Wheeler A, Kouides P, Escobar MA, Leissinger C, Galdzicka S, Corson M, Watson C, Konkle BA. Sood SL, et al. Among authors: ragni m. Blood Adv. 2018 Jun 12;2(11):1325-1333. doi: 10.1182/bloodadvances.2018018226. Blood Adv. 2018. PMID: 29895623 Free PMC article. Clinical Trial.
Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A.
Collins PW, Blanchette VS, Fischer K, Björkman S, Oh M, Fritsch S, Schroth P, Spotts G, Astermark J, Ewenstein B; rAHF-PFM Study Group. Collins PW, et al. J Thromb Haemost. 2009 Mar;7(3):413-20. doi: 10.1111/j.1538-7836.2008.03270.x. Epub 2008 Dec 20. J Thromb Haemost. 2009. PMID: 19143924 Free article. Clinical Trial.
443 results