Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then move in pivotal clinical trials of SerpinPC — an investigational therapy for hemophilia A and B patients, regardless of disease severity or inhibitor status — that it plans to launch…
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In recognition of World Hemophilia Day 2018, the biopharmaceutical company Bioverativ is sharing the stories of people with hemophilia in the developing world to highlight the life-changing potential of adequate treatment. In honor of this year’s theme, “Sharing Knowledge Makes Us Stronger,” Bioverativ will collaborate with members of the hemophilia community…
First in a series. Have you ever wondered what it’s really like to receive gene therapy? I know I have. So when I had the chance to interview 75-year-old Chip Hailey of Tennessee — one of the participants in a clinical trial for Hemgenix (etranacogene dezaparvovec), the first…
I recently bumped into a former colleague at the grocery store. We hadn’t seen each other in several years and enjoyed catching up. (Yes, I was the annoying shopper blocking the aisle.) We had worked together in the healthcare industry, the last place I ever expected to be employed. Working…
BioMarin Pharmaceuticals presented positive interim data from an open-label Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for severe hemophilia A. The promising results, presented at the XXXII International Congress of the World Federation of Hemophilia (WFH), showed that patients had improved and sustained clotting function. BMN 270 is designed to alter…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s developers, announced in a press release. This designation is given to accelerate the development and review of medications…
Workers with hemophilia are at a much higher risk of stroke, developing a joint disease, or needing a knee or hip replacement than the general population, Taiwanese researchers report. The study, “Risk of major comorbidities among workers with hemophilia: A 14-year population-based study,” was published in the journal…
Many hemophilia B patients experience joint pain, depression and overall poor health, a study reports. Other findings were that women with hemophilia B had worse health than men and that those caring for children with the disorder often experience anxiety and depression. The team titled its study in the European Journal…
Hemophilia joint health care involves treatments, physiotherapy, and lifestyle adjustments such as doing low-impact exercises, following a well-balanced nutrition plan, and maintaining good mental health.
My youngest son, Caeleb, is completing his junior year of high school. He’s excited about his senior year and his plans for getting in shape. But there’s something else he’s even more enthusiastic about. Caeleb is beginning to think about life after high school. It may seem late for…