Post by UCL Faculty of Brain Sciences

🧬 We're celebrating a major breakthrough in Huntington’s Disease treatment! For the first time, gene therapy has successfully slowed the progression of Huntington’s disease. 💡 In a landmark trial led by UCL and uniQure, the therapy reduced disease progression by 75% over three years. 🗣️ Professor Sarah Tabrizi, MD PhD FMedSci FRS (UCL Huntington’s Disease Research Centre, UCL Queen Square Institute of Neurology, and UK Dementia Research Institute at UCL), lead scientific advisor on the trial, said: "These groundbreaking data are the most convincing evidence in the field to date and underscore the disease-modifying effect in Huntington's disease, where an urgent need persists. For patients, AMT-130 has the potential to preserve daily function, keep them in work longer, and meaningfully slow disease progression.” 💬 Professor Edward Wild, principal investigator of the UCL Huntington’s Disease Centre trial site at UCL and University College London Hospitals NHS Foundation Trust, said: “This result changes everything. On the basis of these results it seems likely AMT-130 will be the first licensed treatment to slow Huntington’s disease, which is truly world-changing stuff."