Results 41 to 50 of about 13,086,034 (342)

Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis. [PDF]

Clin Pharmacol Ther
In hemophilia B, pharmacokinetic (PK)‐guided dosing of extended half‐life factor IX (EHL‐FIX) concentrates can secure targeted FIX exposure. Target FIX activity levels in plasma should be individually set primarily taking bleeding tendency into account, alongside the presence of target joints, physical activity, and preferred dosing schedules. In other
Koopman SF, Cnossen MH, Mathot RAA, OPTI‐CLOT study group and SYMPHONY consortium.   +3 more
europepmc   +2 more sources

Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A. [PDF]

, 2020
Hemophilia A (HA) is a bleeding disorder characterized by spontaneous and prolonged hemorrhage. The disease is caused by mutations in the coagulation factor 8 gene (F8) leading to factor VIII (FVIII) deficiency.
Agu, Emmanuel, Conroy, Kelsey, Cortez-Toledo, Elizabeth, Gao, Kewa, Hyllen, Alicia A, Nolta, Jan A, Pan, Guangjin, Rose, Melanie, Wang, Aijun, Zhou, Ping   +9 more
core   +1 more source

The history and evolution of the clinical effectiveness of haemophilia type a treatment: a systematic review. [PDF]

, 2012
First evidence of cases of haemophilia dates from ancient Egypt, but it was when Queen Victoria from England in the 19th century transmitted this illness to her descendants, when it became known as the "royal disease".
A Aleem, A Farrugia, A Miners, A Mocroft, AL Dunn, BA Konkle, BL Evatt, BM Feldman, CA Lee, CL Poel Van der, Claudia P. Casas, D Dimichele, D Dimichele, E Berntorp, E Berntorp, E Santagostino, Hector E. Castro, J Astermark, J Goudemand, J Oldenburg, J Stachnik, J Tusell, J Wight, Juan David Rueda, K Porter, L Valentino, L Valentino, LM Aledort, M Carcao, M Carcao, M Depka Von, M Franchini, M Lloyd, M Makris, María Fernanda Briceño, MD Carcao, MV Ragni, P Petrini, PM Mannucci, PM Mannucci, R Prabhu, S Royal, SA Larsson, SC Darby, SC Gouw, T Wong, TL Chorba, VS Blanchette, WK Hoots   +48 more
core   +2 more sources

Long‐term safety and efficacy of rIX‐FP prophylaxis with extended dosing intervals up to 21 days in adults/adolescents with hemophilia B

Journal of Thrombosis and Haemostasis, 2020
An international, multicenter extension study evaluated recombinant fusion protein linking recombinant coagulation factor IX (FIX) with recombinant human albumin (rIX‐FP) in hemophilia B (FIX ≤ 2%) patients previously enrolled in a phase III study or who
M. Mancuso, A. Lubetsky, Brigitte Pan-Petesch, T. Lissitchkov, A. Nagao, W. Seifert, Yanyan Li, E. Santagostino   +7 more
semanticscholar   +1 more source

Assessing bleeding rates, related clinical impact and factor utilization in German hemophilia B patients treated with extended half-life rIX-FP compared to prior drug therapy

Current Medical Research and Opinion, 2020
Objective: An extended half-life factor IX (FIX) fusion protein linking recombinant FIX with recombinant human albumin (rIX-FP), indicated for the treatment of hemophilia B, was approved by the European Medicines Agency in May 2016.
J. Oldenburg, Songkai Yan, G. Maro, G. Krishnarajah, A. Tiede   +4 more
semanticscholar   +1 more source

Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX

Cell Reports, 2018
Summary: Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient ...
Suvasini Ramaswamy, Nina Tonnu, Tushar Menon, Benjamin M. Lewis, Kevin T. Green, Derek Wampler, Paul E. Monahan, Inder M. Verma   +7 more
doaj   +1 more source

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]

, 2003
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM, Nathwani, AC, Ng, CYC, Spence, Y, Zhou, J   +4 more
core   +1 more source

Spontaneous Iliopsoas Hematoma following Microvascular Free Tissue Transfer. [PDF]

, 2017
Spontaneous hematoma within the iliopsoas muscle (SIH) is a rare complication most commonly seen in coagulopathic patients. Often, patients undergoing microvascular free tissue transfer are anticoagulated for anastomotic patency.
Alemi, A Sean, Faden, Daniel L, Heaton, Chase M, Markey, Jeffrey D, Naunheim, Margaret L, Seth, Rahul   +5 more
core   +2 more sources

Haemophilia A: pharmacoeconomic review of prophylaxis treatment versus on-demand [PDF]

, 2015
Objectives. Haemophilia A is a congenital disorder of coagulation that mainly affects males and causes a considerable use of resources, especially when hemophilic patients are treated with prophylaxis.
BOCCIA, Antonio, LA TORRE, Giuseppe, Ricciardi, Walter, UNIM, BRIGID ANDOUNIMYE, Veneziano, Maria Assunta   +4 more
core   +2 more sources

Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

Molecular Therapy: Methods & Clinical Development, 2019
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively.
Benjamin J. Samelson-Jones, Valder R. Arruda   +1 more
doaj   +1 more source