Pfizer will invest an additional $500 million into the construction of its new manufacturing facility in Sanford, North Carolina, the company announced. According to the company, the facility could help advance the manufacturing of one-time gene therapies that use modified adeno-associated viral vectors, such as SB-525, which is…

Mild to moderate hemophilia B affects the relationships of adult patients, children with the disease and their caregivers in aspects such as telling others about the diagnosis, selecting a partner, and feeling bullied, according to an American study. The research, “Mild-severe hemophilia B impacts relationships of…

Although most patients report that discussing their sexual health with their doctors is helpful, hemophilia significantly affects the quality of their sex lives, according to the results of two studies. The research, “Hemophilia and sexual health: results from the HERO and B-HERO-S studies,” was published in…

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…

There is never a time that I do not worry about my sons. “MacDonald the Older” is 23, but not a day goes by that I do not feel concern for his welfare. The phrase “don’t do anything stupid” keeps running through my head. Will I get a call from…

Dear Hemophilia Parent, Today, I write to you. I am a child with hemophilia. I am taking the liberty of speaking on behalf of your child, who may be 2 days, 2 years, or 20 years old. First and foremost, you are awesome, and I…

An opportunity arises Five years ago, Ashley Gregory was asked to head The Female Factor (TFF), a program sponsored by the Hemophilia Foundation of Northern California (HFNC). According to Ashley, this opportunity brought her out of her shell and helped her to discover an affinity and…