25/09/2025
Breakthrough Gene Therapy Offers New Hope for Huntington’s Disease
We are thrilled to share news of a major medical breakthrough in the treatment of Huntington’s disease. A new one-time gene therapy, AMT-130, has shown remarkable results in clinical trials, slowing disease progression by up to 75% over three years. This is the first treatment proven to modify the course of Huntington’s, offering real hope to patients and families affected by this devastating condition.
The therapy works by silencing the faulty gene responsible for producing toxic proteins in the brain. Delivered via a single, carefully guided brain surgery, the treatment has allowed some patients to regain mobility, return to work, and experience a significantly improved quality of life.
At the Neuroprogressive and Dementia Network, we’re proud to support research that transforms lives. This is a huge step forward in neurodegenerative disease research and a powerful reminder of the importance of continued investment in clinical trials and genetic medicine.
A huge step forward—and real hope for families affected.
One of the most devastating diseases finally has a treatment that can slow its progression and transform lives, tearful doctors tell BBC.