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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Manno, Catherine S., Chew, Amy J., Hutchison, Sylvia, Larson, Peter J., Herzog, Roland W., Arruda, Valder R., Tai, Shing Jen, Ragni, Margaret V., Thompson, Arthur, Ozelo, Margareth, Couto, Linda B., Leonard, Debra G.B., Johnson, Frederick A., McClelland, Alan, Scallan, Ciaran, Skarsgard, Erik, Flake, Alan W., Kay, Mark A., High, Katherine A., Glader, Bertil
Published in Blood (15.04.2003)
Published in Blood (15.04.2003)
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Journal Article
Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients
Martinowitz, U., Lissitchkov, T., Lubetsky, A., Jotov, G., Barazani-Brutman, T., Voigt, C., Jacobs, I., Wuerfel, T., Santagostino, E.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.11.2015)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.11.2015)
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Journal Article
Pharmacokinetic study of a high-purity factor IX concentrate (Factor IX Grifols®) with a 6-month follow up in previously treated patients with severe haemophilia B
AZNAR, J. A., CABRERA, N., MATYSIAK, M., ZAWILSKA, K., GERCHEVA, L., ANTONOV, A., MONTAÑÉS, M., PÁEZ, A. M., LISSITCHKOV, T.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.11.2009)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.11.2009)
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Journal Article
Pharmacokinetics, safety and efficacy of a recombinant factor IX product, trenonacog alfa in previously treated haemophilia B patients
Collins, P. W., Quon, D. V. K., Makris, M., Chowdary, P., Kempton, C. L., Apte, S. J., Ramanan, M. V., Hay, C. R. M., Drobic, B., Hua, Y., Babinchak, T. J., Gomperts, E. D.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.01.2018)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.01.2018)
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Journal Article
An open clinical study assessing the efficacy and safety of Factor IX Grifols®, a high-purity Factor IX concentrate, in patients with severe haemophilia B
LISSITCHKOV, T., MATYSIAK, M., ZAWILSKA, K., GERCHEVA, L., ANTONOV, A., MONTAÑES, M., PAEZ, A.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.03.2010)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.03.2010)
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Journal Article
Long-Term Safety and Efficacy of Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Previously Treated Pediatric Patients with Hemophilia B: Results from a Phase 3b Extension Study
Kenet, Gili, Chambost, Hervé, Male, Christoph, Halimeh, Susan, Lambert, Thierry, Li, Yanyan, Seifert, Wilfried, Santagostino, Elena
Published in Thrombosis and haemostasis (01.04.2020)
Published in Thrombosis and haemostasis (01.04.2020)
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Journal Article
Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants
Samelson-Jones, Benjamin J., Finn, Jonathan D., Raffini, Leslie J., Merricks, Elizabeth P., Camire, Rodney M., Nichols, Timothy C., Arruda, Valder R.
Published in Blood advances (09.03.2021)
Published in Blood advances (09.03.2021)
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Journal Article
Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial
Urasinski, T., Stasyshyn, O., Andreeva, T., Rusen, L., Perina, F. G., Oh, M. S., Chapman, M., Pavlova, B. G., Valenta-Singer, B., Abbuehl, B. E.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.03.2015)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.03.2015)
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Journal Article
Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B
Shapiro, Amy D., Kulkarni, Roshni, Ragni, Margaret V., Chambost, Hervé, Mahlangu, Johnny, Oldenburg, Johannes, Nolan, Beatrice, Ozelo, Margareth C., Foster, Meredith C., Willemze, Annemieke, Barnowski, Christopher, Jain, Nisha, Winding, Bent, Dumont, Jennifer, Lethagen, Stefan, Barnes, Chris, Pasi, K. John
Published in Blood advances (11.07.2023)
Published in Blood advances (11.07.2023)
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Journal Article
In silico evaluation of limited blood sampling strategies for individualized recombinant factor IX prophylaxis in hemophilia B patients
Preijers, T., Hazendonk, H. C. A. M., Fijnvandraat, K., Leebeek, F. W. G., Cnossen, M. H., Mathôt, R. A. A.
Published in Journal of thrombosis and haemostasis (01.09.2017)
Published in Journal of thrombosis and haemostasis (01.09.2017)
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Journal Article
Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity
Samelson-Jones, Benjamin J., Finn, Jonathan D., George, Lindsey A., Camire, Rodney M., Arruda, Valder R.
Published in JCI insight (25.07.2019)
Published in JCI insight (25.07.2019)
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Journal Article
Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates
Roth, David A., Kessler, Craig M., Pasi, K. John, Rup, Bonita, Courter, Suzanne G., Tubridy, Karen L.
Published in Blood (15.12.2001)
Published in Blood (15.12.2001)
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Journal Article
Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level ≤2%) haemophilia B
Windyga, J., Lissitchkov, T., Stasyshyn, O., Mamonov, V., Rusen, L., Lamas, J. L., Oh, M.-S., Chapman, M., Fritsch, S., Pavlova, B. G., Wong, W.-Y., Abbuehl, B. E.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.01.2014)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.01.2014)
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Journal Article
Evidence of Multiyear Factor IX Expression by AAV-Mediated Gene Transfer to Skeletal Muscle in an Individual with Severe Hemophilia B
Jiang, Haiyan, Pierce, Glenn F., Ozelo, Margareth C., de Paula, Erich V., Vargas, Joseph A., Smith, Peter, Sommer, Jürg, Luk, Alvin, Manno, Catherine S., High, Katherine A., Arruda, Valder R.
Published in Molecular therapy (01.09.2006)
Published in Molecular therapy (01.09.2006)
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Journal Article
Improvement in health-related quality of life with recombinant factor IX prophylaxis in severe or moderately severe haemophilia B patients: results from the BAX326 Pivotal Study
Windyga, J., Lin, V. W., Epstein, J. D., Ito, D., Xiong, Y., Abbuehl, B. E., Ramirez, J. H.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2014)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2014)
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Journal Article
A retrospective study to describe the incidence of moderate to severe allergic reactions to factor IX in subjects with haemophilia B
RECHT, M., POLLMANN, H., TAGLIAFERRI, A., MUSSO, R., JANCO, R., RICHEY NEUMAN, W.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2011)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2011)
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Journal Article