CAG repeats can get longer over time as the HD gene is used, like the growing potholes and cracks in an old road. New research finds that blocking cells from using their HTT gene slows this wear and tear, which might slow the onset of symptoms in HD.

A new study from Switzerland sheds light on how the CAG repeat number in the Huntington’s disease gene can grow or shrink inside our cells.

UniQure received FDA’s meeting minutes on AMT-130. While there were no new updates, the community’s response has been powerful, with 41K+ petition signatures & unified advocacy from major HD orgs.