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…

My wife, Cazandra, and I will give a presentation titled “Finding Our Way Back to Each Other” at the National Bleeding Disorders Foundation’s upcoming Bleeding Disorders Conference. I invite anyone in the Atlanta area on Friday, Sept. 13, to come and hear what we have to say. The…

Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for adults and children with hemophilia A, according to an analysis of three Phase 3 clinical trials. “These analyses highlight the relevance of pain, physical health, and overall wellbeing as…

My husband, Jared, and I are navigating another significant life transition. Our daughter, Cittie, is now on her second week of kindergarten, which marks the official start of formal schooling here in the Philippines. This transition is even more pronounced because we’ve moved her from a traditional school to a…

I’m trying to accept that my youngest son, Caeleb, is a college freshman. People say that time flies by, and we should enjoy our children when they’re little. Those people are right. As my son begins college, I’m apprehensive. I long for the days when I’d go into school before…

A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis, or preventive therapy, for as long as seven years — the longest follow-up to date for any hemophilia A gene therapy clinical trial. While most of the 12 patients…

What do all the people with bleeding disorders I’ve interviewed for this column have in common? Each of them has a story about certain doctors not listening to them. I have a story about that, too. But first, I want to acknowledge that I now receive exceptional care from…