A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…
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Note: This story was updated March 5, 2026, to correct that tranexamic acid and epsilon-aminocaproic acid prevent the breakdown of blood clots, rather than prevent blood clots. Dental treatments can be performed safely in people with hemophilia when proper medical precautions are followed, according to a review of studies…
This morning I walked with my youngest son, Caeleb, into his school. He depended on a walker to help relieve the pain he felt in his right ankle and knee. The ravages of multiple internal bleeding episodes have left my son with tremendous pain. His ability to walk…
Second in a series. Read part one. The birth of our oldest son, Julian, was a milestone event in our lives. After all the excitement and drama of that stormy June evening in 1996, things calmed down for the rest of the night. My wife, Cazandra, rested in her…
Supporters of World Hemophilia Day, on April 17, are hoping to raise awareness about the bleeding disorder with events planned globally and on social media, and to advocate for sustainable and equitable access to care and treatment. The annual event, which is organized by the World Federation of Hemophilia…
Caeleb is super excited about starting the seventh grade this week. He loves the new town we are living in and is hopeful for the school year. He wants to play basketball. Now, most people would say that is a normal thing for a 12-year-old boy to want…
The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…
Roctavian (valoctocogene roxaparvovec-rvox) is an approved one-time gene therapy that’s intended to reduce the risk of bleeds in certain adults with severe hemophilia A.
Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, with fewer bleeds or breakthrough bleeds, lower costs, and better life quality compared with other extended half-life (EHL) therapies, a modeling analysis suggested. The…
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How Exercise Helps With Hemophilia Management
Prior to the 1970s, people with hemophilia were encouraged to refrain from any form of exercise or physical activity out of fear they would injure themselves. However, the lack of exercise led to obesity and lower bone density and general overall health problems. Nowadays, people living with hemophilia are encouraged to participate in…