Note: This story was updated July 30, 2024, to clarify patients participating in AFFINE had moderately severe to severe disease, and that a secondary study goal involved the mean treated annualized bleeding rate. Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well…

Priscilla Oren, a member of the hemophilia community, recently shared her family’s history with me. Her son was a victim of the “hemophilia holocaust” in the 1980s. During that time, many people with hemophilia contracted HIV/AIDS and hepatitis from the factor products used to treat their bleeding…

Preparing for change is never easy. For Caeleb, my youngest son, this season of his life is stressful yet exciting. After graduating from high school, he is marching on as a freshman at the University of New Mexico this fall. He’s excited about attending college, but has some…

The Canadian Agency for Drugs and Technologies in Health’s (CADTH) Canadian Drug Expert Committee (CDEC) recommended reimbursement for hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec), according to the therapy’s developer, CSL Behring Canada. Hemgenix in 2023 became the first gene therapy in the country approved for hemophilia…

I spent part of a day this week trapped in my condo’s elevator. Since Hurricane Ian hit my town of Punta Gorda, Florida, in September 2022, the elevator has been temperamental, frequently trapping people inside. With high humidity and the outdoor temperature at 98 F, I was hot inside…

The Summer Olympics are around the corner. Gymnasts there will soar through the air with grace and precision, showcasing incredible feats of strength and flexibility. Athletes will push their limits, running at record speeds, demonstrating the peak of human endurance. Divers will climb to dizzying heights, performing acrobatic feats as…

TU7710, an experimental treatment for managing bleeds in people with hemophilia who have inhibitors, demonstrated an average half-life that ranged from 10.4 to 16.6 hours across different doses in a Phase 1a study with healthy volunteers. This extended half-life, that is, the time it takes for a medication’s levels…

Feet and ankle problems are common for people with bleeding disorders, including hemophilia B and von Willebrand disease. As someone living with these conditions, I’m no stranger to these challenges. I recall a trip to Disney World when I decided to wear a pair of Birkenstocks for…

Raising sons with hemophilia is like being a blacksmith forging a sword. The blacksmith shapes a piece of metal that needs careful, skilled effort to become a sharp and strong sword. Each blow of the hammer represents the challenges brought by a bleeding disorder. The process is arduous and…

Hemgenix (etranacogene dezaparvovec), the first approved hemophilia B gene therapy, has been successfully administered to two patients in France, marking the first time the treatment has been given in a real-world setting in Europe. The gene therapy, originally developed by uniQure and marketed by CSL Behring, was…