I am in a video game. My mission: Fight the medical bureaucracy and access care. I must complete three levels and capture the golden key, which unlocks a fortress where healthcare is obtained. I’m stuck on Level 2. No matter what I try, I can’t gather the tools to unlock…

People with hemophilia A in China see major health challenges, such as frequent joint bleeding, joint malformation, and high rates of hospital visits, primarily owing to the limited use of preventive therapies, a real-world study shows. After a bleeding episode, most patients rely on on-demand treatment, which is less…

First in a series. My story starts on a hot and humid night in June 1996 in Houston. As my wife, Cazandra, and I entered the hospital, we couldn’t help but notice the storm gathering outside. We approached the check-in counter, and I explained to the attendant that…

The summer months bring relentless heat. In the desert of New Mexico, where we live, the thermometer often hits triple digits. While some people love the heat, others tolerate it, seeking shelter in the shade of a tree or in an air-conditioned area. Staying cool isn’t just a…

The benefits of a one-time gene therapy were sustained for up to 13 years among men with severe hemophilia B with no new safety concerns emerging, according to newly published results from a clinical trial. The findings, which encompass the longest ever follow-up for a gene therapy study in…

At 6 p.m., Monday through Friday, my television is faithfully tuned to “Jeopardy!” The theme song and sounds of the buzzers usher in a comforting ritual each night that’s part of my home. “Jeopardy!” is a long-running quiz show where contestants are given the answers and must respond with a…

I groggily sat up in the hospital bed, sipped some water, and stared into the distance. Suddenly, a nurse pointed at my gown and shouted, “You’re bleeding!” But I’d taken so many painkillers that I barely reacted. This was 2003, when I had laparoscopic surgery to treat endometriosis.

Last in a series. Read parts one and two. In the final installment of my three-part interview with Chip Hailey, 75, we explore what his life looks like after gene therapy. Chip shares how Hemgenix (etranacogene dezaparvovec) has transformed his daily routine, his mindset, and his outlook…

A lack of family history of disease can delay a diagnosis and timely initiation of preventive therapy in children with hemophilia A, increasing the need for intensive factor replacement therapy when a first bleeding event happens, a study shows. “These observations highlight the importance of an early diagnosis in…