The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…

Oh, my goodness! My youngest son, Caeleb, is preparing for a wonderful senior year of high school. It seems like yesterday my wife and I carried him home from the hospital as a baby and dove into uncharted waters regarding his hemophilia diagnosis. So far in my son’s…

Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in adults with mild or moderate hemophilia A without inhibitors, according to a modeling study. The cost of Hemlibra would need to drop by at least 35% to become cost-effective…

Modern psychology often emphasizes the importance of letting go. In a world constantly pushing us to hold on tightly, learning to let go can be surprisingly liberating. As I get older, I’m realizing the significance of discerning what truly matters in life and cutting out the fluff. Our capacity…

Last Friday, my youngest son, Caeleb, and I started our day as usual and ate at one of our favorite restaurants, Barelas Coffee House in Albuquerque, New Mexico. Summer carries a special magic in the MacDonald home. For many years, I’ve enjoyed spending Museum Fridays with my sons,…

The past two weeks have been especially busy as I produced and directed the musical “Honk! JR.” in my community. Many children who’ve worked with me for years participated in the production. I can’t count the number of children’s theater programs I’ve directed over the years, but I do…

Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.

My husband, Jared, and I have a great connection. In the nine years we’ve been together — five as a married couple — we’ve never run out of things to say. Every day brings a new topic to discuss. Conversation flows easily between us, even if it’s about a controversial…

Nearly two-thirds of children with severe hemophilia A given once-weekly Altuviiio (efanesoctocog alfa) in a Phase 3 clinical trial were completely free of bleeds over about one year of treatment. That’s according to new findings from the recently-completed XTEND-Kids study (NCT04759131), which were presented as late-breaking…

When a spouse or partner has a bleeding disorder like hemophilia, occasionally there may be situations that make us wonder if they are at fault for an injury they’re battling. Perhaps they went beyond their maximum physical capacity in sports or at work. Maybe they got caught up…