Huge leap made in search for Huntington’s disease treatment
- Scientists have achieved a "groundbreaking" milestone by slowing the progression of Huntington's disease for the first time with a new gene therapy.
- The treatment, named AMT-130, is delivered via brain surgery and is expected to last a patient's lifetime after a single dose.
- Early-stage clinical trials involving 29 patients showed that those receiving a high dose experienced 75 per cent less disease progression after 36 months.
- Experts from University College London (UCL), including Professor Ed Wild and Professor Sarah Tabrizi, hailed the findings as potentially "world-changing" and the most convincing evidence to date for a disease-modifying effect.
- The development offers hope for patients, with the potential to preserve daily function, extend working lives, and significantly slow the disease, which currently has no cure.