A one-time gene therapy called AMT-130 has, in topline trial results, slowed Huntington’s disease progression by about 75% over three years—the first disease-modifying signal ever reported for this fatal, inherited condition. The sponsor, uniQure, says it plans an FDA filing in early 2026; full, peer-reviewed data are still pending. The therapy uses an AAV vector … Huntington’s Disease Effectively Treated for the First Time: UK Gene Therapy Trial Achieves ~75% Slower Progression Read More » To access this post, you must purchase Individual Subscriber Yearly or Enterprise Subscriber.