Huntington's disease: Gene therapy shows real hope for slowing brain decline
A clinical trial by uniQure, a US biotechnology company, has tested a new gene therapy, AMT-130, with University College London researchers reporting on the results on people with Huntington's disease—a rare genetic disorder that usually hits in your 30s or 40s.
After three years, patients who got the therapy showed lower levels of a brain degeneration marker and, more importantly, their thinking skills declined more slowly than usual.
Why this matters for the future
This is big news because Huntington's disease typically causes serious movement and memory problems over time.
Another study from UCL and Cambridge also found that subtle changes in thinking can show up years before symptoms get bad—meaning there might be a bigger window to help people early on.
These findings could shape how future treatments are designed and tested.