The ongoing Phase 1/2 Alta trial continues to show that a single infusion of SB-525, an investigational gene therapy, leads to stable and clinically relevant improvements in clotting factor VIII and marked reductions in the need for replacement therapy in patients with hemophilia A. This preliminary follow-up data…

Japan’s Ministry of Health, Labor and Welfare has extended Hemlibra (emicizumab)’s approval to include routine prophylaxis to prevent or lower the frequency of bleeds in people with acquired hemophilia A. Its decision comes seven months after Hemlibra’s maker, Chugai Pharmaceutical, applied for extended use. Hemlibra was previously approved in…

Precision Biologic has announced an update of its Cryocheck Chromogenic Factor VIII assay, a test used to help diagnose and manage hemophilia A. The update, which is expected to be commercially available in the coming months, will ensure that the assay’s accuracy is not compromised by a new…

Life can change in a moment. Sometimes, all it takes is a phone call — that a loved one has passed away, that you’ve lost your job, or that you are free from cancer. Those moments often create core memories — highly emotional, significant experiences that have a lasting…

One of the major hurdles people with hemophilia face is complying with their medication schedule — in particular, injecting themselves several times a week with clotting factor. MORE: Seven recommended books to learn more about hemophilia Clotting factor has a relatively short half-life (the time it takes for a person’s…

My adult son, Julian, does a wonderful job managing his hemophilia. He infuses twice a week per doctor’s orders, and if he has a breakthrough bleed, he treats as needed. Julian is extremely fortunate, because throughout his 24 years, hemophilia has not been the center of his life. His journey…

U.S. hemophiliacs who use patient-reported outcome (PRO) instruments can provide reliable information on their disease burden, finds a new study that appeared in the journal Patient Preference and Adherence. In the study, “Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality…

People with hemophilia A in China see major health challenges, such as frequent joint bleeding, joint malformation, and high rates of hospital visits, primarily owing to the limited use of preventive therapies, a real-world study shows. After a bleeding episode, most patients rely on on-demand treatment, which is less…

I don’t think anyone likes having a chronic disease. I have good days, and I have bad days. Thankfully, the good days outnumber the bad ones. There are days when I tolerate having hemophilia, days when I can step back and see the gifts that have come from…

Health Canada, the agency that oversees health decisions and regulations in Canada, has approved Afstyla (lonoctocog alfa)  for the treatment and prevention of bleeding in adults and children with hemophilia A. In May, the U.S. Food and Drug Administration (FDA) approved Afstyla for the same indications. Developed by CSL Behring, Afstyla is…