For the first time, as part of a U.S. clinical trial, the cell therapy BE-101 has been administered to someone with hemophilia B. Be Biopharma, the company developing BE-101, announced the dosing of the first participant in a first-in-human Phase 1/2 clinical trial called BeCoMe-9 (NCT06611436). The…

Raising two sons with a bleeding disorder has given me hundreds of stories. Many of them lead to happiness and gratitude. Others are filled with pain and suffering. They are all a part of me in a way I never imagined they would be. The nice thing about having so…

A new online service allows patients with bleeding disorders like hemophilia to share important information about their treatment and bleeds with their medical team in real time. MORE: Five tips for living healthily with hemophilia The HemaGo XChange portal was created by Novo Nordisk A/S as an update to their existing website and…

It was the first rain of the season in 1978, and I was in kindergarten. Growing up in a low-income household meant that we were not always prepared, even when my parents wanted to be. I felt fortunate that my mom had a solution to keep my feet dry! She…

The U.S. Food and Drug Administration (FDA) has granted both fast track and rare pediatric disease designations to Expression Therapeutics’ investigational stem cell therapy for hemophilia A. Each of these designations is intended to speed development of a candidate therapy. This stem cell therapy was designed as a potentially…

An infant was diagnosed with severe hemophilia A after developing splenic injury (injury to the spleen), a rare condition among newborns. For the first time, however, a newborn with these conditions was successfully treated with recombinant factor VIII replacement therapy without the need for surgical intervention, doctors reported. The report, titled…

Dear Hemophilia Dad, I am writing to you today to protect the health of your beloved daughter. Trust me, I know the struggles you encounter daily. In my mind, you are a superhero. Your daughter is at risk for hemophilia, too. She is a carrier and needs testing…

A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…

Second in a series. Read part one. A hemophilia inhibitor is like a firewall in a computer network. Clotting factor replacement therapy is supposed to enter the bloodstream and help form clots to stop bleeding, just as data should pass through a network without interruption. However, an inhibitor acts…