Real-world use of the extended half-life factor replacement therapy Elocta (efmoroctocog alfa) was associated with better bleed control than standard half-life products among people with hemophilia A, according to findings from an observational study in Europe. Elocta, sold as Eloctate in the U.S., was also associated with fewer…

Hympavzi (marstacimab-hncq) may be available by prescription in the U.S. before the end of the year. The therapy was approved this month as a routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with hemophilia A or…

Sevenfact (eptacog beta) was deemed safe and effective at stopping bleeds in patients with hemophilia A or B with inhibitors, according to real-world data from a case series study in the U.S. The treatment was also more affordable than other similar therapies and effective even in patients who’d failed…

When the University of the Philippines (UP) unveiled its new motto, “Honor. Excellence. Service.,” this past June, it took me back to my days there as a student. The phrase “Serve the People” reverberates through the campus, reminding every Iskolar ng Bayan (Nation’s Scholar) of…

I have long admired the world-renowned ballerina Michaela DePrince. Like many others, I was captivated by her inspiring journey from being a war orphan in Sierra Leone to her rise as a star on the global dance stage. Tragically, DePrince recently died at the young age of 29, just one…

At 32, Ashley Zebley from Arkansas is no stranger to bleeding disorders. I recently interviewed her over the phone. Ashley explained that her father has severe hemophilia B, and both her maternal grandmother and great-grandmother exhibited bleeding problems, although neither was diagnosed with hemophilia. Ashley herself is a…

MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, has shown promising safety and durability in an ongoing preclinical study in three nonhuman primates, the company has announced. Results showed that after one year, two animals had normal (82%) or nearly normal activity levels (41%) of…

I must admit that, as a father, I dreamed of greatness for my sons. While Cazandra, my wife, was pregnant with each of my boys, I envisioned a global career for them that would transform the world. With my measurement of success as a guiding light, I imagined the…

A team of U.S. researchers has developed a new gene-editing strategy that could make the approach safer and more efficient for treating certain diseases, such as hemophilia. In preclinical studies, the gene-editing technique was shown to enable fast and efficient delivery of healthy genetic material to cells, while bypassing…

My primary care provider recently retired and was replaced by a doctor new to my area. As I prepared for our first meeting, I reflected on how I usually approach my bleeding disorders. Typically, I don’t even mention hemophilia to a primary care doctor. I mutter terms like “bleeding…