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235 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: konkle b. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 Clinical Trial.
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'Osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J. Maguire AM, et al. Among authors: konkle b. N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27. N Engl J Med. 2008. PMID: 18441370 Free PMC article. Clinical Trial.
World Federation of Hemophilia Gene Therapy Registry.
Konkle BA, Coffin D, Pierce GF, Clark C, George L, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I; Members of the WFH Gene Therapy Registery Steering Committee. Konkle BA, et al. Haemophilia. 2020 Jul;26(4):563-564. doi: 10.1111/hae.14015. Epub 2020 May 27. Haemophilia. 2020. PMID: 32462720 Free PMC article. No abstract available.
BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.
Konkle BA, Shapiro AD, Quon DV, Staber JM, Kulkarni R, Ragni MV, Chhabra ES, Poloskey S, Rice K, Katragadda S, Fruebis J, Benson CC. Konkle BA, et al. N Engl J Med. 2020 Sep 10;383(11):1018-1027. doi: 10.1056/NEJMoa2002699. N Engl J Med. 2020. PMID: 32905674 Clinical Trial.
Emerging clinical concerns in the ageing haemophilia patient.
Konkle BA, Kessler C, Aledort L, Andersen J, Fogarty P, Kouides P, Quon D, Ragni M, Zakarija A, Ewenstein B. Konkle BA, et al. Haemophilia. 2009 Nov;15(6):1197-209. doi: 10.1111/j.1365-2516.2009.02066.x. Epub 2009 Jul 21. Haemophilia. 2009. PMID: 19686466 Review.
Rituximab for treatment of inhibitors in haemophilia A. A Phase II study.
Leissinger C, Josephson CD, Granger S, Konkle BA, Kruse-Jarres R, Ragni MV, Journeycake JM, Valentino L, Key NS, Gill JC, McCrae KR, Neufeld EJ, Manno C, Raffini L, Saxena K, Torres M, Marder V, Bennett CM, Assmann SF. Leissinger C, et al. Among authors: konkle ba. Thromb Haemost. 2014 Sep 2;112(3):445-58. doi: 10.1160/TH14-01-0078. Epub 2014 Jun 12. Thromb Haemost. 2014. PMID: 24919980 Free PMC article. Clinical Trial.
Von Willebrand factor for menorrhagia: a survey and literature review.
Ragni MV, Machin N, Malec LM, James AH, Kessler CM, Konkle BA, Kouides PA, Neff AT, Philipp CS, Brambilla DJ. Ragni MV, et al. Among authors: konkle ba. Haemophilia. 2016 May;22(3):397-402. doi: 10.1111/hae.12898. Epub 2016 Feb 4. Haemophilia. 2016. PMID: 26843404 Free PMC article. Review.
235 results