My husband, Jared, and I are just starting to figure out family life. As novices to the world of parenting and homemaking, we’re navigating our child’s formative years and learning to maintain a household through trial and error. Living by ourselves has been an especially helpful experience. Choosing not…

The end of 2022 is rapidly approaching, and I’ve made nary a dent in my fitness and diet goals for this year. Oh, sure, I have a ton of excuses — like my foot that swells up when I walk more than a mile — but there really…

Treatment with marstacimab (PF-06741086), an experimental antibody-based therapy developed by Pfizer, reduced the number of bleeds in patients with severe hemophilia who took part in a Phase 1b/2 clinical study. This held true regardless of whether patients had hemophilia A or hemophilia B, or had inhibitors…

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…