News

Researchers, working with mice, have developed a new way of producing substantial numbers of gene-corrected liver cells with the possibility of repopulation in vivo. The strategy, which relies on the liver’s unusual regenerative skills, may hold promise for the treatment of diseases such as hemophilia B. The study by scientists at Oregon Health & Science University,…

Researchers at the University of Tokyo reviewed the status of physical activity among hemophilia patients to discuss strategies to encourage activity through a behavioral change approach by focusing on hemophilia-relevant factors such as benefits and bleeding risk, risk management of bleeding, physical activity characteristics, and difficulty in exercise adherence. The study,…

uniQure N.V., a leading pharmaceutical specialized in human gene therapy, has presented additional data from its clinical trial in hemophilia B patients at the 21st Congress of the European Hematology Association (EHA),  June 9-12, in Copenhagen. The Phase 1/2 clinical trial of AMT-060 titled “Trial of…

A recent clinical trial indicates that replacement therapy with plasma-derived factor VIII is associated with lower incidence for development of inhibitors in patients with severe hemophilia A. The study,“A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,” was published in The New England Journal of…

Researchers at the Birjand University of Medical Sciences and colleagues investigated the immunity status of hemophilic patients against hepatitis A, and the need for hepatitis A vaccination in that patient population. Findings indicate that a significant proportion of hemophilic patients under age 20, who were evaluated in the study, had no immunity…

  The difference in disease severity between Hemophilia A and B (HA, HB) was recently assessed in the study, “Clinical, Instrumental, Serological And Histological Findings Suggest That Hemophilia B May Be Less Severe Than Hemophilia A,” and published in Haematologica. HA and HB are caused by deficient clotting proteins,…

Facial and scalp swelling can be indicators of childhood hemophilia, but for children who are misdiagnosed and treated incorrectly, the effects of the disease’s elevated bleeding predisposition can be catastrophic. In the study, “Facial and Scalp Swelling in the Pediatric Population With Hemophilia: A Diagnosis Pitfall,” published in…

The safety and efficacy of emicizumab in patients with severe hemophilia A (HA) were recently evaluated in a study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” published in The New England Journal Of Medicine. Hemophilia A is caused by a lack of blood clotting factor…

Chugai Pharmaceutical recently published data of a Phase I clinical study on the antibody emicizumab. The study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” was published in The New England Journal of Medicine. The study revealed possible benefits from emicizumab  for the prevention of bleeding in…

Sports and physical activity can benefit people living with hemophilia, according to a recent study. Researchers in Germany and the United Kingdom investigated to find whether participating in sports can benefit adults with varying degrees of hemophilia severity. The report, “The impact of sport on health status,…