The U.S. Food and Drug Administration (FDA) has agreed to review uniQure’s application seeking accelerated approval of AMT-130, a gene therapy candidate for Huntington’s disease that is now called ifezuntirgene inilparvovec. “The FDA’s acceptance of our [application] for review marks an important milestone for the Huntington’s disease community,”…
Discussion
My wife, Jill, who is gene-positive with Huntington’s disease (HD), and I often talk about what we owe the future stewards of the healthcare system. It weighs on us as a family affected by a rare disease. When you spend a lot of time in doctors’ offices and waiting…
Discussion
There is something powerful about standing in a room and telling the truth about what it means to live with Huntington’s disease (HD). Every time I share my story, I am reminded that advocacy is not just about speaking. It is about being seen, making space, and helping change…
Discussion
The experimental therapy brezosiran can reduce levels of the mutant huntingtin protein in people with Huntington’s disease, according to interim data from a small clinical trial. The results were shared in oral and poster presentations at the European Huntington’s Disease Network Clinical Research Congress 2026, held Oct.
Becoming educated and informed about Huntington’s disease 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 Huntington’s disease journey.
There is no cure for Huntington’s disease 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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