Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B. The goal is to advance the company’s hemophilia B program…

Pfizer is preparing to launch its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) after securing a U.S. approval for the treatment last month. Beqvez is the second gene therapy to be approved for adults with moderate to severe hemophilia B in the U.S., following the clearance of…

Problems at work, limited engagement in recreational activities, and less relationship satisfaction contribute to a lower health-related quality of life (HRQoL) and greater pain severity, as reported by adults with hemophilia B, according to a study. Findings also showed that anxiety and depression in caregivers of children with this…

CSL Behring marked World Hemophilia Day April 17 by shipping more than 10 million international units (IUs) of its medicines to treat hemophilia A to developing countries through the nonprofit World Federation of Hemophilia (WFH). The medicines were sent to countries like Vietnam, Cameroon and others through the WFH’s…

The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog beta for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors. A…

Easter Sunday was wonderful. The pipe organ at our church hadn’t worked in four years, but thanks to generous donors, it was finally restored. We revealed it on Easter with a brass quintet and choir, and the music was truly beautiful. As a classically trained oboist, I am deeply…

“Use the Press’n Seal to cover his bandages when showering!” That’s the text I sent to the wife of a hospitalized friend, who was preparing to be released after an arduous stay. Hearing of his plight with doctors, nurses, and a second surgery was reminiscent of past hospitalizations with my…

A conversation Last night I dreamed that I spoke with my father. I asked him how he knew he had a bleed. Did his knee bubble like a soda? Was it tight, but not painful? I yearned for his input, but my alarm sounded before he was able…

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,…

Service dogs are typically thought of as necessary companions for the visually impaired, but service and therapy dogs can be a practical solution for people with a variety of chronic illnesses. As well as being a trusted friend, service dogs can expand owners’ motor abilities, granting them new independence and allowing them…