Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a small interview study assessing patient perceptions. These patients reported that they choose their treatments based mainly on five criteria. Specifically, those are…

Recent advances in gene therapy approaches to treat hemophilia B were recently announced by uniQure. The company achieved “substantial progress in advancing our lead program in hemophilia B,” Matthew Kapusta, CEO of uniQure, said in a press release. The company reacquired the development and commercial rights to…

My family gathered to take pictures. We drove to the Bosque, the cottonwood forest that borders the Rio Grande and runs through New Mexico. It’s a beautiful place with running water and trees. The photographer took numerous action shots, one of which brought a reality check. The photographer had us…

A telerehabilitation program, wherein physical therapy exercises were delivered to patients by a professional via video calls, was deemed safe and effective at improving the physical condition of people with severe hemophilia, according to a small study in Chile. Patients reported a high degree of satisfaction with the intervention,…

First in a series. Have you ever wondered what it’s really like to receive gene therapy? I know I have. So when I had the chance to interview 75-year-old Chip Hailey of Tennessee — one of the participants in a clinical trial for Hemgenix (etranacogene dezaparvovec), the first…

In our quest for security and certainty, it’s only natural to gravitate toward options that offer zero risk. Think about it: When you purchase insurance or opt for a product with a money-back guarantee, you’re essentially seeking assurance against any potential loss. This tendency to avoid risk at all…

Last in a series. Read parts one and two. For 21-year-old Domenic Catrine, hemophilia B once dictated every part of life, from wearing a helmet during childhood playtime to college routines shaped by weekly factor infusions. Then he underwent gene therapy during his sophomore year in…

I have diabetes and give myself insulin shots every evening. There is not much to the process. I draw up the insulin into a syringe and find a place in the area around my stomach. Once I choose the site, I plunge the tiny needle into my skin. No harm,…

Novo Nordisk has submitted an application to the U.S. Food and Drug Administration seeking approval to market Mim8 (denecimig), an injection therapy for preventing bleeds in people with hemophilia A with or without inhibitors. If approved, Mim8 would be sold as a single-use, prefilled disposable pen for subcutaneous,…