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Page 1
Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients.
Shapiro AD, Ragni MV, Valentino LA, Key NS, Josephson NC, Powell JS, Cheng G, Thompson AR, Goyal J, Tubridy KL, Peters RT, Dumont JA, Euwart D, Li L, Hallén B, Gozzi P, Bitonti AJ, Jiang H, Luk A, Pierce GF. Shapiro AD, et al. Among authors: josephson nc. Blood. 2012 Jan 19;119(3):666-72. doi: 10.1182/blood-2011-07-367003. Epub 2011 Nov 22. Blood. 2012. PMID: 22110246 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: josephson nc. 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: josephson nc. 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-acting recombinant factor IX Fc fusion protein (rFIXFc) for perioperative management of subjects with haemophilia B in the phase 3 B-LONG study.
Powell JS, Apte S, Chambost H, Hermans C, Jackson S, Josephson NC, Mahlangu JN, Ozelo MC, Peerlinck K, Pasi J, Perry D, Ragni MV, Wang X, Jiang H, Li S, Cristiano LM, Innes A, Nugent K, Brennan A, Luk A, Allen G, Pierce GF, Robinson B. Powell JS, et al. Among authors: josephson nc. Br J Haematol. 2015 Jan;168(1):124-34. doi: 10.1111/bjh.13112. Epub 2014 Sep 11. Br J Haematol. 2015. PMID: 25208598 Free article. Clinical Trial.
Results of genetic analysis of 11 341 participants enrolled in the My Life, Our Future hemophilia genotyping initiative in the United States.
Johnsen JM, Fletcher SN, Dove A, McCracken H, Martin BK, Kircher M, Josephson NC, Shendure J, Ruuska SE, Valentino LA, Pierce GF, Watson C, Cheng D, Recht M, Konkle BA. Johnsen JM, et al. Among authors: josephson nc. J Thromb Haemost. 2022 Sep;20(9):2022-2034. doi: 10.1111/jth.15805. Epub 2022 Jul 17. J Thromb Haemost. 2022. PMID: 35770352 Free article.
Novel approach to genetic analysis and results in 3000 hemophilia patients enrolled in the My Life, Our Future initiative.
Johnsen JM, Fletcher SN, Huston H, Roberge S, Martin BK, Kircher M, Josephson NC, Shendure J, Ruuska S, Koerper MA, Morales J, Pierce GF, Aschman DJ, Konkle BA. Johnsen JM, et al. Among authors: josephson nc. Blood Adv. 2017 May 18;1(13):824-834. doi: 10.1182/bloodadvances.2016002923. eCollection 2017 May 23. Blood Adv. 2017. PMID: 29296726 Free PMC article.
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.
Konkle BA, Walsh CE, Escobar MA, Josephson NC, Young G, von Drygalski A, McPhee SWJ, Samulski RJ, Bilic I, de la Rosa M, Reipert BM, Rottensteiner H, Scheiflinger F, Chapin JC, Ewenstein B, Monahan PE. Konkle BA, et al. Among authors: josephson nc. Blood. 2021 Feb 11;137(6):763-774. doi: 10.1182/blood.2019004625. Blood. 2021. PMID: 33067633 Free PMC article. Clinical Trial.
Pilot randomized, non-inferiority, cross-over trial of once-weekly vs. three times-weekly recombinant factor VIII prophylaxis in adults with severe haemophilia A.
Ragni MV, Yabes JG, Fogarty PF, Josephson NC, Kessler CM, Neff AT, Raffini L, Brummel-Ziedins K, Moore CG. Ragni MV, et al. Among authors: josephson nc. Haemophilia. 2017 Jan;23(1):e43-e46. doi: 10.1111/hae.13131. Epub 2016 Dec 11. Haemophilia. 2017. PMID: 27943502 Free PMC article. Clinical Trial. No abstract available.
38 results