The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…

Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in adults with mild or moderate hemophilia A without inhibitors, according to a modeling study. The cost of Hemlibra would need to drop by at least 35% to become cost-effective…

Last Friday, my youngest son, Caeleb, and I started our day as usual and ate at one of our favorite restaurants, Barelas Coffee House in Albuquerque, New Mexico. Summer carries a special magic in the MacDonald home. For many years, I’ve enjoyed spending Museum Fridays with my sons,…

Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.

Nearly two-thirds of children with severe hemophilia A given once-weekly Altuviiio (efanesoctocog alfa) in a Phase 3 clinical trial were completely free of bleeds over about one year of treatment. That’s according to new findings from the recently-completed XTEND-Kids study (NCT04759131), which were presented as late-breaking…

I spent some of the best years of my life at Galena Park High School in Texas. I participated in clubs only if they didn’t interfere with my work with band, and my grades were excellent. When I met my now husband in college, our drive to…

Incidences of bleeding from playing sports are rare among people with hemophilia, and more likely to occur in those with lower levels of clotting factors — proteins that play a key role in promoting blood clotting — a study in the Netherlands has found. More specifically, the study reveals…

My happy place is in front of an audience. It’s where I feel most comfortable. As I’m a self-proclaimed introvert, that may sound odd. Yet I’ve had numerous opportunities to answer my call to speak. It’s not about accolades and praise; it’s about the message. Regardless of the size of…

Note: This story was updated May 8, 2023, to remove hemophilia as one of the specific indications the collaboration will focus on, as these have not yet been publicly disclosed. AstraZeneca and Sernova are teaming up for a preclinical research project that aims to develop new cell therapies…

2seventy bio announced that it has achieved a key preclinical milestone under its joint program with Novo Nordisk to develop a gene-editing tool to treat hemophilia A. Details of the “proof of concept” research that met this milestone — reported to be worth $15 million to the…