Caeleb, the child doctors said I’d never have, has changed my life. He is 16 and thinking about his future. That may sound like a normal thing for a 16-year-old, but I was unsure about his future with his hemophilia A. His life with this disease has been…

The effectiveness of liver-directed, viral-based gene transfer — the most used experimental gene therapy approach for hemophilia A — is significantly reduced in mouse models of hemophilia A relative to healthy mice, a study shows. This limitation was associated with a pronounced reduction in fenestrations — cell surface…

Katherine High, MD, has received the gold medal from Children’s Hospital of Philadelphia (CHOP) in recognition of her work to advance gene therapies for hemophilia and other genetic disorders. In CHOP’s 166-year history, this award — to recognize the most significant achievements in improving the health of children —…

Roche has extended its partnership with the World Federation of Hemophilia (WFH) to expand access to treatments for hemophilia through the end of 2028, the company announced. Under the partnership, Roche will continue to provide its prophylactic (preventive) treatment for hemophilia A to the WFH’s Humanitarian…

A blood test to determine the amount of active factor IX — the clotting factor that is missing in hemophilia B — in patients ages 2 and older is now available in clinical labs in Canada, the European Union, Australia, and New Zealand. The test, called CRYOcheck Chromogenic…

Treatment with abatacept, a medication approved for rheumatoid arthritis, prevented the formation of inhibitors against hemophilia A replacement therapies in a rat model, a study demonstrates. These findings support the use of abatacept to prevent immune responses against human proteins in a preclinical setting, the researchers noted. The study,…

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on the Phase 3 trial testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy for hemophilia A. Pfizer, which is developing the therapy along with Sangamo Therapeutics, expects to begin dosing…

Eptacog beta was safe and almost 100% effective at stopping bleeds within 24 hours in boys ages 12 and younger with hemophilia A or B, according to final data from the Phase 3 PERSEPT 2 trial. According to researchers, this bypass agent may be an important option for…

Believe Limited, continuing a partnership with BioMarin Pharmaceutical and working with the Foundation for Art & Healing, announced plans to produce a documentary film on the everyday challenges and victories of living with a bleeding disorder. The digital content production company, again with the support of BioMarin, produced “…

Factor VIII (FVIII) levels in the bloodstream of men with severe hemophilia A treated with the investigational gene therapy Roctavian appear to be influenced by regulatory molecules involved in F8 gene activity and FVIII protein folding and release, a study reveals. Researchers also found a dose-dependent relationship between…