Small vesicles in the saliva of people with severe hemophilia A may explain why these patients rarely have bleeds in their mouth and throat. The vesicles contain a protein complex that’s able to induce blood coagulation in these patients, thereby reducing bleeding. The findings may help “develop innovative approaches…

For the first time in Denmark, people with hemophilia B have been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to an announcement from CSL Behring, the company that markets the therapy. Hemgenix was cleared for use in the European Union in early 2023, following its…

I don’t recall when I met Debbie Murray, but I remember the impression she left on me. Her frustration with the long and challenging journey to a proper diagnosis and effective treatment of her hemophilia was unmistakable. Debbie, 56, resides in Norwood, a village in the northernmost part of…

Be Biopharma has raised $92 million in series C financing to support the first-in-human Phase 1/2 trial of BE-101, the company’s B-cell therapy candidate for hemophilia B. Proceeds from the funding round will be partly used to generate proof-of concept data from the BeCoMe-9 trial (NCT06611436), which…

When I tell people I’m a woman with hemophilia, I’m often met with the same response: “Well, that is really unusual. It’s rare for women to have hemophilia!” This ubiquitous misconception is the bane of every female hemophiliac’s existence. It often stands in the way of women and…

Two new patients in the U.S. have received Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B, per new reports. The therapy was first administered last year in May to a patient at UC San Diego Health, per a University of California, San Diego, news…

I have to admit, I was captivated by Hemophilia News Today‘s Facebook page during the final days of December, eagerly following its countdown of the year’s top 10 most-read columns. It was an incredible honor to see that my columns secured eight of those 10 spots. Each one…

The start of a new year often brings a surge of enthusiasm, hope, and resolutions. We might set ambitious goals like, “This is the year I lose weight.” Or, “This is the year I go to the gym five days a week.” Or, “This is the year I mend a…

Me: “Hello?” Medical assistant: “Hi, Shellye, the doctor wants to see you ASAP to discuss your recent test results. She’s concerned.” Me: “Oh no, that doesn’t sound good. Can you tell me what’s going on?” Medical assistant: “We just got your baseline bone density tests back and they show you…

Sangamo Therapeutics will regain full rights to develop and market giroctocogene fitelparvovec, a gene therapy candidate for adults with moderately severe to severe hemophilia A, after Pfizer decided to end its involvement in the program. Pfizer’s decision not to advance into regulatory submissions came despite positive…