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 ...
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 ...
A new Huntington’s disease (HD) clinical trial has reached an important milestone: the first participants have now been dosed in POINT-HD, a Phase 1 study testing a new, selective huntingtin-lowering ...
One mystery that many scientists think holds the key to curing HD is its mysterious age of onset. Although people with HD carry the expanded gene from birth, they generally don’t develop symptoms ...
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 ...
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 ...
The one-year report from the TRACK-HD study demonstrates a number of changes in pre-symptomatic HD-mutation carriers ...
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