A collaborative research study offers new insights into how a protein called furin influences blood clotting and found it functions differently in hemophilia A and B, findings that are promising for improving gene therapy for hemophilia A. The study, “Circumventing furin enhances factor VIII biological activity and ameliorates bleeding…

I’m a big fan of the stage version of “Wicked.” In the opening of the second act, Glinda (the “Good”) is standing in front of the citizens of Oz, sharing her delight in being center stage. She expresses her humblest gratitutions (you’ll understand why I’m using that strange…

Save One Life, the first globally focused organization to sponsor children with bleeding disorders one-on-one, recently celebrated its 15th anniversary. More than 130 guests from the hemophilia community worldwide gathered in New York City Sept. 29 at the Manhattan Penthouse on Fifth Avenue. The guest of honor was…

Novo Nordisk has entered into a collaboration agreement with Bluebird Bio to develop new therapies based on gene editing for a series of genetic disorders, including hemophilia. The companies have agreed to work together on a three-year research project that will focus on the identification and…

When my boys were little, I dreamed about what their chosen activities might be — band, swimming, theater, or maybe soccer. I wanted to be a soccer mom, cheering on my sons from the sidelines and bringing orange slices for the team. My oldest son, Julian, did play soccer…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B. This designation is given to therapies that are meant to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. It offers…

Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the activity of clotting factor VIII (FVIII) are continuing in all five severe hemophilia A patients treated at the highest dose in the Alta Phase 1/2 trial. None of these…

Researchers at several Canadian institutions investigated the tendencies of health-related quality of life (HRQoL) among teenagers and young adults with hemophilia A (HA) and found that young adults demonstrated more joint disease and worse HRQoL when physical function and pain were considered. The research paper, “Generic and disease-specific quality of…

In contrast to observations in the general population, blood type and levels of von Willebrand factor (VWF) don’t appear to influence factor VIII (FVIII) levels in people with non-severe hemophilia A, a study reports. Moreover, age was found to play only a minor role in FVIII levels in…

New swimming trunks, sunscreen, cap, sleeping bag — gathering these items means it’s time for hemophilia camp! My son Caeleb is so excited that he is driving his dad and me a little crazy. Two years ago, he broke his wrist the night before camp and wasn’t…