As we prepare to celebrate Christmas, I can’t help but remember a particular year in the MacDonald household. In 2013, my youngest son, Caeleb, couldn’t catch a break from hemophilia. Constant spontaneous bleeding episodes into his right knee and ankle kept my boy in the hospital more often…

Proteins from tardigrades — microscopic animals known for their ability to survive in extreme conditions — could be used to stabilize replacement therapies for hemophilia A, allowing treatments to be stored for longer periods of time without refrigeration, a proof-of-concept study shows. “Our work provides a proof of principle…

February 2014 was a significant time in the life of my family. For years we saw our younger son regularly suffer bleed after bleed. He suffered from an inhibitor and an allergy to factor. Nothing seemed to help. We felt lost and unsure about how to continue seeking…

San Rafael, California-based BioMarin Pharmaceutical will present interim data of an open-label, Phase 1/2 clinical trial of BMN 270, an investigational gene therapy treatment for hemophilia at the upcoming XXXII International Congress of the World Federation of Hemophilia (WFH) July 24-28 in Orlando, according to a news release. The late-breaking…

Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then move in pivotal clinical trials of SerpinPC — an investigational therapy for hemophilia A and B patients, regardless of disease severity or inhibitor status — that it plans to launch…

When I was 3 years old, I joined a television pageant for little girls called “Princess Asia.” I won the top prize. But when I look back on that moment now, the crown itself feels almost incidental. What stands out more is that I caught amebiasis, an intestinal infection, just…

I’ve been pondering the minimalism movement a lot lately. This is because I recently found myself with more than a dozen boxes from my childhood home, and along with these came a sense of responsibility to organize the contents inside of them. As my husband and I unpacked the…

There is a funny cartoon making the Facebook rounds. It shows two images, one for “normal parents” and the other for “medical field parents.” The normal parents’ side has a duck dressed as a doctor and a mama duck holding her baby up, saying, “He sneezed! Help…

Preventive treatment with Eloctate (efmoroctocog alfa) for up to five years led to sustained reductions in all bleed types for children, adolescents, and adults with severe hemophilia A, according to data from two Phase 3 trials and their open-label extension study. New analyses of these pivotal trials also…

Last week, I was privileged to attend Genentech‘s Rare Blood Disease Summit at its campus in San Francisco. I’ve been proud to serve on this biotechnology company’s Women in Hemophilia advisory board for the past two years. The invitation-only summit was a small but powerful gathering, bringing together the…