Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A that combines Genevant’s proprietary lipid nanoparticle (LNP) platform with cutting-edge mRNA-based megaTAL technology. The partners will seek to advance a treatment involving gene editing — used to correct, add, or delete…
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When my youngest son, Caeleb, was born over 19 years ago, my wife and I found out that he had hemophilia before leaving the hospital. Devastated, we could not begin to tell anyone how we felt about the diagnosis. I remembered our struggles raising my oldest son, Julian, now…
There’s a saying that’s been on my mind a lot lately: “Action is the antidote to anxiety.” It resonates deeply, especially in our household, where uncertainty is part of daily life. My husband, Jared, has severe hemophilia B and epilepsy — two conditions that bring unique challenges. Together, they…
Why is this survey being conducted? The purpose of this survey is to gather information from individuals with hemophilia so that Bionews Services and its partners can bring better understanding, advocacy, and provide our readers more relevant editorial content to the larger hemophilia community. What will I do? If you…
Some words can be offensive. I’m not talking about some of the obvious, obnoxious words in the world, I am talking about two words in particular: disease and disorder. If you look at a list of rare diseases, hemophilia is listed. But you don’t ever hear anyone say,…
When my first son, Julian, was born in 1996 and diagnosed with severe hemophilia A, I thought my world was ending. I expected to give birth to a healthy baby boy. I never dreamed he’d have a medical condition. I went through the stages of grief: denial,…
At every annual event sponsored by the National Hemophilia Foundation, a particular room is dedicated to remembering those who died from complications of hemophilia. In the 1980s and early ’90s, many people lost loved ones during a period that became known as the “hemophilia holocaust.” A staggering number…
This week, my youngest son, Caeleb, visited the pediatric clinic for his last comprehensive exam there. My mind went in a million directions as I recalled meeting his hematologist, Shirley Abraham, MD. She witnessed the tough times when my son struggled through many painful spontaneous bleeding episodes because…
My son Caeleb is 14 and entering high school this fall. He is at an age in which I, as a parent, do not speak on his behalf in front of him. He is old enough to share his story about his hemophilia diagnosis when he deems it appropriate. I…
Children and young adults with hemophilia A and hemophilia B have several behavioral problems, ranging from depression and anxiety to aggressive behaviors, a small study suggests. Age, disease severity, and joint disease duration were found to be significantly correlated with several of these problems. The findings were reported in the…