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…

My sons, Julian and Caeleb, are adults living with hemophilia. Julian is 28 years old, and Caeleb is 18. Having adult children is a new phase of life for me. While Julian is out on his own chasing the next audition, Caeleb is preparing to begin…

Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…

I think about my daughter’s future a lot, especially now that she’s getting older. At 5 and a half, she’s just finished senior nursery and is headed for big school in a month. She’s skinny and nimble, having lost most of her baby fat except for her cheeks. She loves…

Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B and SPK-8011 (dirloctocogene samoparvovec), an experimental treatment for hemophilia A. LLUH’s Center of Excellence Hemophilia Program is structured to ensure that financial constraints don’t prevent access to the therapy,…

I love sitting by a campfire. Stories and laughter are shared, s’mores and hot dogs are roasted, and memories are made. Tending the fire to keep the flames burning requires constant attention, and it takes my mind off my troubles. Watching the flames leap and dance is mesmerizing. However, the…

Note: This article was updated June 26, 2024, to clarify that the analysis of fitusiran in surgery was conducted in patients receiving prophylactic treatment with the therapy. The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults…

I first met Kim Phelan, chief operating officer of The Coalition for Hemophilia B, in person at a recent symposium in Dallas. From the moment we spoke, I was in awe of her. Despite the hundreds of attendees, she took the time to engage with me personally.

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…