Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for adults and children with hemophilia A, according to an analysis of three Phase 3 clinical trials. “These analyses highlight the relevance of pain, physical health, and overall wellbeing as…
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I’m trying to accept that my youngest son, Caeleb, is a college freshman. People say that time flies by, and we should enjoy our children when they’re little. Those people are right. As my son begins college, I’m apprehensive. I long for the days when I’d go into school before…
A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis, or preventive therapy, for as long as seven years — the longest follow-up to date for any hemophilia A gene therapy clinical trial. While most of the 12 patients…
In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy. The company has struggled to turn a profit from the gene therapy, which was approved in…
As a woman of faith, I look for God in various places. I experience God in a beautiful sunset, a phone call from a dear friend, or even in the middle of a struggle. Walking a labyrinth is one of my favorite places to connect with God. A labyrinth…
Game shows have experienced a resurgence in recent years, captivating audiences with their blend of entertainment, competition, and nostalgia. I’ve always liked game shows, especially those involving trivia. In “Who Wants to Be a Millionaire?,” contestants answer multiple-choice questions; if they’re correct, they advance to the next level, with…
Nearly two-thirds of children with severe hemophilia A on preventive, or prophylactic, treatment with once-weekly Altuviiio (efanesoctocog alfa) in the yearlong XTEND-Kids study were free of bleeding episodes needing treatment. More than 80% in this Phase 3 clinical trial also were free of spontaneous bleedings and joint bleeds needing…
When my sons with hemophilia were children, my role as their advocate seemed clearly defined. My wife, Cazandra, and I sought as many sources as possible to make the best decisions for their welfare, consulting leading specialists to help us when complications arose and spontaneous bleeding…
The past two weeks have been a whirlwind of activity for me, highlighted by two significant events. First, I attended an event in Washington, D.C., called “Voices of Hemophilia,” which was sponsored by the biotech company CSL Behring. This small but powerful gathering brought together eight leading voices from…
Most caregivers or patients with hemophilia who were on preventive treatment were satisfied or very satisfied with the Florio Haemo smartphone app, even after prolonged use, a survey study suggests. The app lets patients track their infusions, bleeds, pain, and activities, and helps them manage their medication stock.