Hemophilia A, a deficiency of functional coagulation factor VIII (FVIII), is treated via protein replacement therapy. Restoring 1% to 5% of normal blood FVIII activity prevents spontaneous bleeding, making the disease an attractive gene therapy target. Previously, we have demonstrated short-term activity of a liver-specific AAV2 vector expressing canine B-domain-deleted FVIII (cFVIII) in a hemo...

Background: Mothers and fathers mentioned difficulty in identifying the proper structure of remedy for their child, anxiousness about illness blood products, fear achievable long-term aspect consequences prophylaxis, a desire to avoid any unnecessary medications. It is clear from what has been noted above that mother appreciably want health care program help hemophilic young people limit compli...

Intravenous administration of recombinant human factor IX (rhFIX) acutely corrects the coagulopathy in hemophilia B dogs. To date, 20 of 20 dogs developed inhibitory antibodies to the xenoprotein, making it impossible to determine if new human FIX products, formulations, or methods of chronic administration can reduce bleeding frequency. Our goal was to determine whether hemophilia B dogs rende...

UNLABELLED BACKGROUND & OBJECTIVE  The factor VIII (FVIII) B domain shares very little amino acid homology with other known proteins and is not directly necessary for procoagulant activity. Despite this, missense mutations within the B domain have been reported in patients with hemophilia A. Given that the B domain is dispensable for secretion and function of FVIII, we hypothesized that thes...

Introduction Epidemiologic studies suggest that joint bleeding occurs in patients with mild-to-moderate haemophilia, including women and girls. However, most previous on the impacts of haemophilia focus men severe disease. Aim To identify unmet needs haemophilia. Methods The Pain, Functional Impairment, Quality Life (P-FiQ) study assessed impact pain functional impairment health-related quality...

We gave danazol (600 mg/day orally for 14 days) to eight adults with mild or moderate hemophilia A, one with severe hemophilia A, and one with moderate hemophilia B. In the patient with severe hemophilia A, the levels of factor VIII two to four days after an infusion of factor VIII concentrate were higher than expected, suggesting a prolonged half-life. In one patient with mild hemophilia A, a ...

the pediatric risk of mortality (prism) score is a measure of illness severity based on abnormalities observed on bedside examination and laboratory assessment at pediatric intensive care unit (picu) admission to predict mortality probability. our study was performed on 205 patients who were admitted to picu of children’s medical center (cmc) over a period of 6 months.data were recorded prospec...

background rheumatoid arthritis (ra) is a chronic inflammatory disease with extra- articular involvement. eyes in particular are one of the most common sites of involvement in rheumatoid arthritis. on the other hand, dry eye syndrome is the most common conflict in terms of management. particular studies suggested dry eye has been associated with greater rheumatoid arthritis severity, but its as...

Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or IX genes. The palliative treatment of choice based on the use safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable durable concentrations defective circulating factor. Results have so far been encouraging in terms levels times expression using mainly adeno-associate...

Hemophilia is a serious rare disease that requires continuous management and treatment for which the medicine is costly at the annual average of 100 million KRW for an individual. The aim of this study was to investigate trends in the utilization of coagulation factor (CF) used for hemophilia treatment using the National Health Insurance database from 2010 to 2013 in Korea and compare the utili...