European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…

Dear Newly Diagnosed Sister, Welcome! You are now part of a supportive community full of love, courage, and inspirational superheroes. Always remember that you are not alone. I was diagnosed with von Willebrand disease and hemophilia B in 1975, at age 10. Your diagnosis Diagnosis of…

An innovative gel containing two snake venom proteins known to help blood clotting, rapidly and effectively forms stable clots and reduces bleeding even in the presence of a blood-clotting disorder, as is the case in hemophilia, a preclinical study shows. The gel, which transitions to a liquid form below…

Last week I attended the New Mexico Annual Conference of the United Methodist Church. As a pastor, I must attend the conference every year. One positive aspect of the meeting is a focus on clergy health. A medical assistant measured me, took my blood pressure, weighed me, and took a…

My 16-year-old son, Caeleb, is on summer break. He told me that summer is a little too boring and the school year is a little too much. He is definitely a teenager. Next month he begins summer band, and boredom will become exhaustion. Caeleb is already concerned about marching…

It’s normal to come across inconveniences in daily life. Imagine being in the following scenarios: You’re filling out an important document with a pen when it runs out of ink midsentence. It’s a typical morning and you’re on your regular train to work when it breaks down out of…

ASC Therapeutics has joined forces with Charles River to scale the manufacturing of ASC618, its second-generation virus-based gene therapy for hemophilia A. The agreement expands upon their current collaboration, begun in 2019 that focused on achieving Good Manufacturing Practice (GMP)-virus manufacturing and establishing processes for adeno-associated virus (AAV) production…

As my COVID-19 recovery continues, I have questions. So I called public health expert Frank James, MD, to help. James is active in teaching, research, and medical practice. He is the health officer for San Juan County and the Nooksack Indian Nation in Washington state. He is…

Last week, my 16-year-old son, Caeleb, had a physical therapy session at 1 p.m. Unfortunately, the business of the day prevented us from visiting a museum. I felt disappointed that I didn’t hold up my end of the bargain regarding our plans for regular Friday “museum days” during…

Dosing has begun in a second group of hemophilia B patients enrolled in a Phase 1/2 dose-confirmation trial of the experimental gene therapy FLT180a, the treatment’s developer, Freeline, announced. The dose determined to be optimal in this study is expected to finalize plans for a pivotal Phase 3…