The one-time gene therapy BBM-H901 (dalnacogene ponparvovec) has been officially approved in the Macao region of China for the treatment of adults with moderate to severe hemophilia B, thus expanding access to the medication beyond mainland China. Developed by Belief Biomed (BBM), BBM-H901 became China’s first and only…
Discussion
When people think about hemophilia, they usually think of joint, muscle, and sometimes soft-tissue bleeds. But one of the most dangerous complications occurs elsewhere: the brain. My husband, Jared, had a brain bleed as a child. It’s the kind of event people often imagine as…
A small survey study in Germany found that many people with severe hemophilia are not interested in receiving gene therapy, despite having “only general knowledge” of the treatment option — which, the researchers noted, could be “a potential cure with a single infusion.” According to the scientists, these…
Discussion
It started with a refrigerator leaking water out of the bottom and a YouTube video that made it seem like an easy fix. You know the kind: “Just remove the freezer door.” “Melt the ice with a hair dryer.” “You’ve got this.” It didn’t seem like a big deal, but…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
Becoming educated and informed about hemophilia is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your hemophilia journey.
There is no cure for hemophilia yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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