Note: This story was updated March 28, 2024, to clarify the gene therapy delivers the mRNA of the Cas9 enzyme. Intellia Therapeutics is ending its collaborative agreement with Regeneron, which had the two companies working together in developing hemophilia B gene-editing therapies, according to a securities and exchange…

Every week, I lead the pastoral prayer at church. It’s a moment to share what brings us joy and what weighs on our hearts as a congregation and a community. I always pray for the unhoused, the hungry, the grieving, and our country. But there’s one group I often mention…

When you’re a parent to a child newly diagnosed with a bleeding disorder, the immediate concern is for your child. What do I do next? Will they be OK? What will life look like from now on? You find yourself searching for answers and developing a support system…

A single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox) continues to prevent bleeds and the need for preventive treatment (prophylaxis) for up to five years in most treated men with severe hemophilia A, according to final Phase 3 trial results. Activity of factor VIII (FVIII), the clotting…

The cost of the one-time hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) will now be covered for eligible patients in Germany through the country’s public healthcare system. This comes through a reimbursement price agreement between developer CSL Behring and GKV-Spitzenverband, the German National Association of Statutory Health…

I’m a 25-year-old “PWD” (person with disabilities), and I’m not a baby bird anymore. It’s time for me to fly on my own, be independent, and take care of myself. Recently, I had a string of bleeding episodes. It was difficult for me to put a smile on my face.

Fat-shaming is a widespread issue that can also affect people with hemophilia, especially young children. At its core, fat-shaming involves making negative comments or judgments about a person’s weight, which can lead to feelings of inadequacy or embarrassment. While these remarks may be intended to encourage weight loss or…

Dear pharmaceutical companies,  I am speaking to every one of you who makes a factor replacement product for hemophilia.  Thank you for developing more options and safer products for our community. Because of your research, we have products that greatly improve our lives. We…

People whose hemophilia A is mild or moderate can show marked alterations to their joints on MRI scans, particularly in the ankles, despite having few joint bleeds, a study reported. These findings highlight the need for closer monitoring of joint health and a likely need for preventive treatment approaches in patients…

On Sunday mornings in my home, I usually watch “CBS Sunday Morning,” hosted by Jane Pauley. Last week, an interview caught my eye. The segment featured Dr. Jennifer Doudna, who won the Nobel Prize in chemistry in 2020 along with fellow researcher Emmanuelle Charpentier for developing…