A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the use of clotting factor VIII (FVIII) for up to four years in men with hemophilia A, according to the latest data from a Phase 1/2 trial and its extension study.

When my youngest son was 4, he wanted to play baseball. My nerves were shaky as I took him to register for the fall season. I hoped he would enjoy playing the sport. When the first game of the season rolled around, my wife and I sat in the bleachers,…

My son Caeleb begins his sophomore year of high school in a few weeks. Before school starts, he will participate in summer band. Summer band was a highlight of my high school days. Even marching outside in the heat was fun because working with a tightknit group to put on…

Two years of preventive treatment with Genentech’s Hemlibra (emicizumab) safely and effectively lowered the number of bleeds in people with hemophilia A and clotting factor VIII (FVIII) inhibitors, according to final data from the Phase 3b STASEY trial. Development of antibodies against the therapy (anti-drug antibodies) was…

When it comes to home maintenance and improvement, there are several types of people. Some look the other way when their home needs upgrades due to a lack of time, money, energy, or interest. Others coordinate with repair people who come in and do the necessary work. Do-it-yourself people look…

I’ve been sick for most of this week with digestive issues. This is a chronic condition for me, but it always bothers me when it happens. I’m weak and unable to do much. I must gauge my energy levels and try to squeeze important tasks into the moments I…

People with severe hemophilia A who switch from standard to extended half-life replacement therapies experience fewer bleeds and require less frequent infusions, an analysis of patients in Austria indicates. Whether these gains are reflected in lower overall treatment costs, however, is less clear, its researchers added. Data from this…

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…

My youngest son, Caeleb, is embarking on a new adventure and studying for his driver’s test. Once he earns his permit, we’ll take him to the church parking lot for practice. Caeleb is thrilled at the thought of driving to school and band rehearsals. He is growing up and realizing…