The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical’s new application seeking approval of its investigational one-time gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. The application was resubmitted to EMA following a request for longer follow-up data from the Phase 3 GENEr8-1 clinical trial (NCT03370913),…

“MacDonald the Older,” which I call my eldest son, successfully auditioned for the International Performing Arts Institute, a national musical theater symposium. He calls me nearly every night to share the great things he learned in different classes. My heart leaps up and down with joy when I hear his…

One of the good things about being married to a person with disability (PwD) is that I can talk to my husband, Jared, objectively about disability and chronic illness. We often have conversations about it, and we aren’t afraid of self-criticism. In fact, we value it for the sake of…

Preventive treatment with Hemlibra (emicizumab) sustainably improved physical health-related quality of life and led to fewer missed workdays among people with severe hemophilia A without inhibitors, an analysis of pooled data from two clinical trials found. According to researchers, these findings add to previous data showing that Hemlibra…

For boys and men with moderate to severe hemophilia, switching to extended half-life (EHL) products is linked to a reduced number of infusions and a sustained low rate of bleeds, according to a  real-life, multi-center study in Canada. Notably, a significant drop in the annualized bleeding rate (ABR)…

BioMarin Pharmaceutical has resubmitted a regulatory application seeking approval in Europe for its experimental gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. That resubmission, to the European Medicines Agency (EMA), included one-year follow-up data from BioMarin on the therapy’s safety and effectiveness in treating the rare genetic…

Less-frequent and easier administration of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — is likely to boost treatment adherence among patients with severe disease, a South Korean study has found. The study, “Patients’ and parents’…

I received an email from an old friend. She remembers when my youngest son, Caeleb, was in and out of the hospital. We both have experience raising a son with hemophilia and an inhibitor. Our paths are terribly similar, and seeing her name pop up in my email was…

My dear husband, I won’t do everything for you. I may take on some of your physical responsibilities when your body says “no for now,” but I’ll leave you to do all the thinking and strategizing you can do for our family. You may have hemophilia and a seizure disorder,…

Joint pain significantly rose and overall joint health — particularly range of motion — diminished with limitations on physical activities during the COVID-19 lockdown for people with hemophilia and joint disease, a small study from Spain reported. The study “Musculoskeletal Changes in Hemophilia Patients Subsequent to COVID−19…