Plenary Paper CLINICAL TRIALS AND OBSERVATIONS Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
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چکیده
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A Johnny Mahlangu, Jerry S. Powell, Margaret V. Ragni, Pratima Chowdary, Neil C. Josephson, Ingrid Pabinger, Hideji Hanabusa, Naresh Gupta, Roshni Kulkarni, Patrick Fogarty, David Perry, Amy Shapiro, K. John Pasi, Shashikant Apte, Ivan Nestorov, Haiyan Jiang, Shuanglian Li, Srividya Neelakantan, Lynda M. Cristiano, Jaya Goyal, Jurg M. Sommer, Jennifer A. Dumont, Nigel Dodd, Karen Nugent, Gloria Vigliani, Alvin Luk, Aoife Brennan, and Glenn F. Pierce, for the A-LONG Investigators
منابع مشابه
CLINICAL TRIALS AND OBSERVATIONS Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients
Current factor VIII (FVIII) products display a half-life (t1/2) of 8-12 hours, requiring frequent intravenous injections for prophylaxis and treatment of patients with hemophilia A. rFVIIIFc is a recombinant fusion protein composed of a single molecule of FVIII covalently linked to the Fc domain of human IgG1 to extend circulating rFVIII t1/2. This first-in-human study in previously treated sub...
متن کاملCLINICAL TRIALS AND OBSERVATIONS Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study
1Van Creveldkliniek, University Medical Center Utrecht, the Netherlands; 2Department of Pediatrics, Wilhelmina Children’s Hospital, University Medical Center Utrecht, Utrecht, the Netherlands; 3Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands; 4Prof Hess-Kinderklinik, Zentrum fur Kinderheilkunde und Jugendmedizin, Bremen, Germany; 5Centre of Pediatr...
متن کاملCLINICAL TRIALS AND OBSERVATIONS Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients
1Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre, Istituto di Ricovero e Cura a Carattere Scientifico Cà Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy; 2Centre Régional de Traitement de l’Hémophilie, Hôpital Edouard Herriot, University Claude Bernard, Lyon, France; 3Haemophilia Treatment Centre, Vivantes Klinikum im Friedrichshain, Vivantes Hospital, Berlin, Germany; 4He...
متن کاملRegular Article CLINICAL TRIALS AND OBSERVATIONS Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011
Department of Haematology, University Hospital of Wales, School of Medicine, Cardiff, United Kingdom; The UK National Haemophilia Database, Manchester, United Kingdom; Department of Haematology, Royal Hospital for Sick Children, Glasgow, United Kingdom; The Haemophilia Centre, The Royal London Hospital, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, Unit...
متن کاملSustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector.
The successful prophylactic treatment of hemophilia A by frequent infusions of plasma concentrates or recombinant factor VIII (hFVIII) indicates that gene therapy may be a potential alternative for the treatment of the disease. For efficient delivery and long-term expression of the hFVIII gene, a novel minimal adenovirus (mini-Ad) vector, MiniAdFVIII, has been developed. The vector is devoid of...
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