Clinical trials for hemophilia are essential to advance medical knowledge and develop treatments. But for decades, trials related to hemophilia enrolled only men, given the assumption that women were only carriers of the disease. Consequently, women with the disorder were denied the opportunity to participate in…

First in a series.  Treatment for people living with hemophilia has long meant a lifetime of infusions, careful planning, and constant vigilance. But new therapies are rewriting what’s possible, particularly gene therapy. Domenic Catrine is a charismatic 21-year-old who lives with severe hemophilia B. A junior in college…

A liver transplant can be live-saving for hemophilia patients, but may not always fully cure the disease, according to the case report about a hemophilia A patient. The letter to the editor describing the report, “Orthotopic liver transplantation for haemophilia A may not…

Roche is initiating a Phase 3 clinical trial to test the experimental gene therapy SPK-8011 in people with hemophilia A. The company announced the move in its 2023 half-year results. At the same time, however, Roche is discontinuing the development of another investigational hemophilia A gene therapy…

A single infusion of Beqvez (fidanacogene elaparvovec-dzkt), a now-discontinued gene therapy for hemophilia B, led to sustained production of factor IX (FIX) — the clotting protein missing in people with the condition — for up to six years, reducing bleeding episodes and eliminating the need for routine preventive…

Researchers at the Center for Research in Transplantation and Immunology in France have reviewed a paper concerning certain strategies utilized to correct hemophilia B and the importance of gene delivery vehicles in successful therapies for treatment of the condition. The review paper, “Successful correction of hemophilia by CRISPR/Cas9 genome…

To help people understand the science behind hemophilia and other bleeding disorders, digital content agency Believe Limited will present its first science fair this fall. Sponsored by commercial gene therapy company Spark Therapeutics, the science fair will be held at the National Hemophilia Foundation’s 71st Bleeding Disorders Conference…

When I am asked how Thanksgiving was for my family, I tell people that my little family ate a feast at home, watched television and relaxed. It was uneventful, nothing exciting. And that is exciting in itself. When you live with a bleeding disorder, your life can be…

Novo Nordisk has entered into a collaboration agreement with Bluebird Bio to develop new therapies based on gene editing for a series of genetic disorders, including hemophilia. The companies have agreed to work together on a three-year research project that will focus on the identification and…

In the marketing world, we have a concept called the elevator pitch. An elevator pitch is a short, persuasive speech delivered in the time it takes to ride an elevator, hence the name. The goal of an elevator pitch is to quickly and effectively communicate a person’s ideas, product, or…