Hemophilia B is an X-linked hemorrhagic disease affecting about 1/30,000 males. The disease manifests as unpredictable, recurrent, and spontaneous bleeding in soft tissues and/or major joints. This recurrent bleeding disease is due to a defect in the blood coagulation factor IX (FIX). Recent ...
For example: An infant with hemophilia should have appropriate padding in his crib or playpen. A toddler with hemophilia should be supervised as he learns to walk. A school-age child with hemophilia should avoid contact sports and activities that have a high risk of trauma. ...
the ability of B-lymphoid progeny of transduced HSCs to induce tolerance and direct sustained therapeutic levels of FVIII in hemophilia A mice, we have shifted our focus to mature antigen-specific memory B cells (MBCs) as an alternative target cell population for gene therapy of hemophilia A. ...
Use of factor XIIa for treatment of hemophilia A and B and prevention of bleedingThe present invention is directed to a Factor XIIa-coated solid support comprising a recombinant Factor XIIa polypeptide or a Factor XIIa-expressing cell comprising an expression vector construct containing a gene ...
The term is most commonly used to refer to two specific conditions known as hemophilia A and hemophilia B, which will be the main subjects of this article. Hemophilia A and B are distinguished by the specific gene that is mutated (altered to become defective) and codes for a defective ...
Figure 2. In vivo efficacy of AAV-mediated gene transfer of cFVIIa in hemophilia A and B dogs. (A) AAV8 vector used for infusion in hemophilia mice and dogs. Inverted terminal repeats (ITRs) flank the expression cassette composed of an hAAT/apoE promoter/enhancer, a synthetic intron, the...
ADVATE® [Antihemophilic Factor (Recombinant)] is a treatment for hemophilia A, a factor VIII deficiency. See Detailed Important Risk Information and full Prescribing Information.
READY TO MOVE ON? SEVENFACT For the treatment of bleeding episodes in people* with hemophilia A or B with inhibitors SEVENFACT Life Stories Learn how SEVENFACT has helped make patients' lives more predictable. THE FIRST INHIBITOR BYPASSING AGENT APPROVED IN OVER 20 YEARS ...
Beqvez, which is given as a single intravenous infusion, was shown in a clinical trial of 45 people to be better at preventing bleeding among adults with moderate to severe hemophilia B compared to regular infusions of a protein that promotes clotting, called protein factor IX (FIX). The cu...
An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B. Journal of blood medicineN. Machin, M.V. Ragni, An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B, J. ... Nicoletta,Machin,V Margaret,... - 《...