Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well tolerated and superior to standard replacement therapy in reducing bleeds, according to new top-line results from a Phase 3 clinical trial. The open-label trial, dubbed AFFINE (NCT04370054) and sponsored by Pfizer, has now treated 75 men with moderate to severe disease. […] The post Hemophilia A gene therapy superior to FVIII replacement in Phase 3 trial appeared first on Hemophilia News Today ..read more

Priscilla Oren, a member of the hemophilia community, recently shared her family’s history with me. Her son was a victim of the “hemophilia holocaust” in the 1980s. During that time, many people with hemophilia contracted HIV/AIDS and hepatitis from the factor products used to treat their bleeding disorders. As a result, Oren’s daughter has become […] The post Addressing medical trauma in the hemophilia community appeared first on Hemophilia News Today ..read more

Preparing for change is never easy. For Caeleb, my youngest son, this season of his life is stressful yet exciting. After graduating from high school, he is marching on as a freshman at the University of New Mexico this fall. He’s excited about attending college, but has some anxiety about what the future holds. Caeleb […] The post Preparing for college means putting hemophilia in its place appeared first on Hemophilia News Today ..read more

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 the potential to earn more money. If they answer every question correctly, they win a $1 million prize. Contestants also have lifelines they may use when stumped by a question. They can poll the audience, have two wrong answers eliminated from their c ..read more

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 treatment. Joint bleeds, a common issue in hemophilia A, can contribute to joint problems. Full trial results were published in The New England Journal of Medicine in the study, “Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A.” Full d ..read more

I recently stumbled upon a thought-provoking post by “Typical Pinoy Crap” (TPC), a well-known Filipino digital creator who shares insightful views about social issues. The post commented on a line from a song by the all-girl Filipino pop group BINI, titled “Karera” (“Race”). The line is “Huwag magalala buhay ay ‘di karera,” meaning “Don’t worry, life isn’t a race.” TPC’s reply caught my attention: “Yes, life isn’t a race. But just because you’re not racing doesn’t mean you should walk when you have the capacity to run.” He elaborated that while life isn’t about constant competition, it’s cruc ..read more

The Canadian Agency for Drugs and Technologies in Health’s (CADTH) Canadian Drug Expert Committee (CDEC) recommended reimbursement for hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec), according to the therapy’s developer, CSL Behring Canada. Hemgenix in 2023 became the first gene therapy in the country approved for hemophilia B when the federal regulatory agency Health Canada approved it to treat adults 18 and older with hemophilia B who need routine prophylaxis to prevent or reduce the frequency of bleeding episodes. The CDEC said its recommendation to make Hemgenix eligi ..read more

I spent part of a day this week trapped in my condo’s elevator. Since Hurricane Ian hit my town of Punta Gorda, Florida, in September 2022, the elevator has been temperamental, frequently trapping people inside. With high humidity and the outdoor temperature at 98 F, I was hot inside the elevator. But I’d followed the advice I give my elderly neighbors: “Always have your phone with you when you ride the elevator.” I usually add “and carry water,” but I was only going to retrieve my mail, so I didn’t have any with me. The elevator had made a loud noise and then stopped. I then called 911, and ..read more

Cazandra and Caeleb, my wife and youngest son, rode with me several years ago to see a movie in Albuquerque, New Mexico, near our home. Caz and I planned the whole day with one goal: to have fun. After the movie, we’d have lunch and then go to a mall. But on the trip, as I used my blinker to exit the interstate, a car attempted to make its way onto the freeway illegally. Without a way to avoid a collision, I crashed into the vehicle, which pushed my dashboard back toward my wife and me. I drove to the side of the road as I watched the other car leave the scene. A big dent between the driver’s ..read more

The Summer Olympics are around the corner. Gymnasts there will soar through the air with grace and precision, showcasing incredible feats of strength and flexibility. Athletes will push their limits, running at record speeds, demonstrating the peak of human endurance. Divers will climb to dizzying heights, performing acrobatic feats as they somersault and twist before slicing into the water’s depths. Each event is a testament to years of dedication, training, and perseverance. Spectators are privileged to watch these tremendous athletes participate in the most important competition of their l ..read more