Scientists engineered stem cells with “interrupted” CAG repeats to break up the toxic stretch. This may stop expansion, and could improve problems in cells that model Huntington’s disease. This study ...
POINT-HD has begun dosing its first participants with the drug RG6496, marking an early but important step for a new selective huntingtin-lowering approach.
CAG repeats can get longer over time as the HD gene is used, like the growing potholes and cracks in an old road. New ...
When it comes to thinking about the effects of Huntington’s disease (HD), most people automatically start to think about the brain due to the severe symptoms caused by the breakdown of brain cells. It ...
On December 4, 2025, uniQure announced they have received the final meeting minutes from their October 29 pre-Biologics License Application (BLA) meeting with the FDA regarding AMT-130. The minutes ...
Living with HD is challenging enough, but depression and anxiety, two of the most common psychiatric symptoms in HD, can make everything exponentially harder. Imagine trying to navigate your daily ...
Ever since large genetic studies in Huntington’s disease (HD) revealed that the longer the CAG expansion, the earlier symptoms appear, we’ve known that repeat length matters. Recent work has ...
In plain language. Written by scientists. For the global HD community. A new study from Switzerland sheds light on how the CAG repeat number in the Huntington’s disease gene can grow or shrink inside ...
In plain language. Written by scientists. For the global HD community. A new study from Switzerland sheds light on how the CAG repeat number in the Huntington’s disease gene can grow or shrink inside ...
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