I live in the middle of nowhere. It is one of the most beautiful places on the planet. The forest and the ocean intersect, creating some of the most gorgeous hiking trails I have had the pleasure of traversing. Nature is our playground, with thousands of acres of beaches, forests,…

SerpinPC, an experimental treatment being developed by Centessa Therapeutics, continues to be safe when used for up to nearly three years and to almost zero out the number of bleeds in adults with severe hemophilia A or B. Those findings come from a one-year extension of the…

Hemlibra (emicizumab-KXWH) safely reduces bleeding episodes in people with hemophilia A without inhibitors, resulting in a high rate of patient satisfaction, according to a review of published studies. “The success of [Hemlibra] underscores the potential for innovative therapeutic approaches to significantly improve patient outcomes and quality of life…

I am lucky to live in the United States, where medication to treat hemophilia and von Willebrand disease is easy to obtain. Had I been born in a developing country, I might not be alive today. My treatment includes injections of expensive blood-clotting medicine called factor. Access to…

Healthcare costs can be 25 times higher for hemophilia B patients than for individuals who do not have a bleeding disorder, according to a recent study. These findings reflect both direct costs of hemophilia treatment and a greater need for other healthcare resources, researchers noted. The study, “…

The National Institutes of Health (NIH) awarded the DNA Medicine Institute (DMI) a $1.5 million grant to advance a quick and easy way of monitoring factor VIII (FVIII) and Hemlibra (emicizumab) blood levels in people with hemophilia A, especially those in developing countries. DMI,…

A new study highlights a hidden emotional burden for families living with hemophilia: the deep sense of guilt felt by parents, particularly mothers, who pass the condition on to their children. However, researchers found that this weight can be significantly lifted through better access to medical information, professional counseling,…

Durveqtix (fidanacogene elaparvovec), a gene therapy for adults with severe and moderately severe hemophilia B, has been granted conditional marketing authorization by regulators in the European Union. The decision by the European Commission follows a positive recommendation on the …

Hemophilia A patients who do not refrigerate their bleeding prevention therapy are more satisfied with it and report fewer restrictions on their daily activities, a study finds. Researchers also discovered that most of the patients were unaware that they could keep their recombinant factor VIII clotting therapy at room temperature.

Several years ago, I spoke at a great bleeding disorders conference in California. Around 300 people attended, and we all had wonderful interactions. The attendees engaged with one another and found hope in their shared experiences. I left the conference energized and proud to be part of such a…