A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) prevents bleeds and the need for preventive treatment, or prophylaxis, for up to six years in men with severe hemophilia A, according to updated data from a Phase 1/2 trial. “With every year of observation in this…

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…

A simple tonsillectomy led to my eventual diagnosis of hemophilia B and von Willebrand disease. Without a family history, a bleeding disorder wasn’t on my family’s or pediatrician’s radar. I had to almost bleed to death for a bleeding disorder to be suspected. Terror of the tonsils The…

The gene therapy BBM-H901 increased factor IX activity in 10 men with moderate to severe hemophilia B, while reducing bleeds and the use of FIX replacement therapy over one year, according to results of a pilot Phase 1 trial. “The study was the first clinical trial conducted by Belief BioMed…

Many times when my sons were younger, I found myself overwhelmed by the constant demands of their bleeding disorder and felt that I had nothing else to give. The needle on my gas gauge was below empty. I felt as if I’d used every ounce of energy and couldn’t…

Spring is in the air. With flowers blooming and birds chirping, this time of year is filled with new life and new beginnings. It’s also graduation season, a time that brings hope. Families share loved ones’ accomplishments and celebrate the end of one season and the beginning of the next.

The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for EtranaDez (etranacogene dezaparvovec), an investigational gene therapy for adults with hemophilia B. The FDA grants priority review to investigational therapies designed to treat serious medical conditions. If approved, the treatment…

When I was younger, my family moved around a lot. I also spent a lot of time traveling all over the Philippines with my mom, due to her government job. I especially loved going to the beach, whether to swim or just listen to the soothing sound of the waves…

One of the unexpected issues I struggled with when my youngest son, Caeleb, faced many prolonged hospital stays due to internal bleeding episodes was loneliness. My wife, Cazandra, and I divided our time between the hospital and home life with our older son, Julian. As a result, we…

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…