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Break Up With Your CAGs: How Three Letters Could Change Huntington’s Disease
Huntington’s disease (HD) is caused by a repeated stretch of the genetic letters C-A-G within the huntingtin (HTT) gene above a critical number. If the…
First participants dosed in new POINT-HD huntingtin-lowering trial
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…
A road less traveled: how making less huntingtin can alter somatic instability and may delay symptoms
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…
A tug-of-war at the DNA: how Huntington’s repeats grow and shrink
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…
UniQure Receives FDA Meeting Minutes on AMT-130 While Community Support Remains Strong
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…