News

Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…

Preventive treatment with Hemlibra (emicizumab) is superior to replacement therapy with factor VIII infusions for reducing bleed rates in people with hemophilia A without inhibitors, a pooled analysis of trials shows. The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A…

Genetic variations in certain immune-related genes, namely the HLA and IL-10 genes, are linked with an increased risk for developing inhibitors against factor VIII replacement therapies in patients with severe hemophilia A. The study that determined that finding, “Risk stratification integrating genetic data for factor VIII inhibitor development…

The prevalence of hypertension is higher in patients with hemophilia than in the general population, according to a nationwide study in Sweden with long-term follow-up. In contrast, no differences were seen in the prevalence of angina pectoris and myocardial ischemia, two cardiovascular diseases. The study, “…

Mitsubishi Tanabe Pharma is joining forces with researchers from Jichi Medical University to launch a program in Japan focused on developing a gene therapy product to treat patients with hemophilia B, the company announced. The project, titled “R&D of gene therapy…

The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) asked Bayer to clarify the risks to the hemophilia community, and the potential effects, of the recent voluntary recall of two defective lots of Kogenate FS anti-hemophilic factor. In a letter sent to Paul…

The presence of inhibitors and excessive body weight increase the sense of disease burden felt by parents of children with moderate to severe hemophilia, a study reports. The study “Burden on parents of children with hemophilia: the impact of socio- demographic and child’s medical condition” was published in…

To encourage more women to take part in disease research, the Patient-Centered Outcomes Research Institute (PCORI) has chosen the Hemophilia Federation of America (HFA) and its FIRST project to receive a Eugene Washington PCORI Engagement Award. The HFA’s FIRST project stands for Females in Research Sharing and…

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…

Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…