If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient compared with other preventive treatments, specifically Hemlibra. That’s according to a draft evidence report issued by pricing watchdog the Institute for Clinical and Economic Review (ICER). An independent non-profit…

My husband, Jared, and I are currently in the process of looking for preschools for our daughter, Cittie. She’s almost 4, but sometimes she acts and speaks as if she’s much older. Cittie often tells us that she wants to go to school. Although we refuse to rush her…

This morning, I took my 16-year-old son, Caeleb, to physical therapy at Carrie Tingley Hospital in Albuquerque, New Mexico. He continues to experience recurring pains from past internal bleeding episodes in his right knee and ankle. The goal is to find the source of the pain and a…

A participant in a Phase 3 clinical trial evaluating Roctavian (valoctocogene roxaparvovec), a gene therapy for hemophilia A, has been diagnosed with leukemia. A genetic assessment of the case, conducted by the therapy’s developer, BioMarin Pharmaceutical, suggested the cancer diagnosis was not linked to Roctavian. Details of…

Hemophilia A patients who develop inhibitors — neutralizing antibodies made by immune cells against clotting factors — have more expression, or activity, of genes involved in activating the immune system, a small study reports. “The results of our study reveal that there is an upregulation of genes involved with…

Last week, I visited an outstanding church member in a local hospital. When I arrived, I saw a friend in the lobby, looked into her eyes, and knew that something was dreadfully wrong. She explained that her baby suffered unexplained medical issues and required immediate care. Parents whose children are…

Shared decision-making in healthcare was a topic at the National Hemophilia Foundation’s Bleeding Disorders Conference I attended virtually last week. Shared decision-making means you have a voice and your overall well-being should be considered as part of any treatment plan. We think of medicine as a science. But as…

As I entered my son’s bedroom this morning to turn on his light and wake him up, I heard him say a phrase that sent shivers down my spine. Caeleb, my boy, said, “Dad, my ankle hurts.” My first instinct wanted to allow him extra time to get himself together.

A recent news story caught my attention. The report highlighted local athletes who unfortunately sustained injuries while serving in the military. These amazing women and men play basketball, tennis, and several other sports while in wheelchairs. Watching these phenomenal veterans play with determination and the physical prowess of professional athletes…

Good thing this column isn’t a podcast because I’m not speaking today. Last night, I chomped down on my tongue for the second time in two months. Not a big deal, you might say. True. Unless you have a bleeding disorder like hemophilia B, von Willebrand disease, or,…