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…

My husband, Jared, and I have maintained a small garden on our balcony ever since we moved into our new home. We started out with just a few potted herbs, and as time went by, we expanded our collection to include some ornamental plants and flowers. Jared is a wizard…

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 —…

It’s easy to spot physical damage, such as swollen joints, nosebleeds, limps, and bruises, when one suffers from a bleeding disorder. But the psychological and emotional damage that may result is not as easy to see or treat. A U.S. survey of hemophilia patients, published a few years…

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…

I love the final-night event of a national bleeding disorder conference. Different groups invite community members to play games, the meal is remarkable, and the dance floor begs for us all to gather and celebrate. After the highly informative events offered by leaders in the bleeding disorder community, friends…

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…

Affirmation: one word with significant connotations. From the day we come into this world, we seek an acknowledgment that our presence on this planet matters. To be affirmed by those around us reinforces the idea that our contributions to our community hold special significance. When we hear others give…