SPK-8011 for hemophilia
SPK-8011, also known as dirloctocogene samoparvovec, is an experimental gene therapy for hemophilia A. Given as a one-time intravenous or into-the-vein, infusion, it aims to reduce the risk of bleeds in patients.
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Gene therapy Durveqtix OK’d for use in Europe for hemophilia B
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 …
How life skills training could help people with hemophilia
My husband, Jared, and I have been working on some big home renovations lately. Under the supervision of my dad, who is a civil engineer, Jared learned how to do various jobs. He rerouted some water pipes, plugged up some holes, and painted. He also watched as an electrician installed…
Addressing medical trauma in the hemophilia community
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 college means putting hemophilia in its place
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…
CADTH recommends hemophilia B gene therapy reimbursement
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…
Reexamining hemophilia emergency protocols and the need for advocacy
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…
Passing the baton to a new team in my son’s hemophilia care
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…
TiumBio’s hemophilia bypassing agent shows extended half-life
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…
Supportive footwear are essential for managing my hemophilia
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…