Groundbreaking Hope for Once Untreatable Disease

Scientists have unveiled the first-ever treatment that makes a real impact on Huntington’s disease — a genetic neurodegenerative disorder once seen as untreatable.

In a groundbreaking trial, researchers achieved a 75% slowing of disease progression over three years.

In the study, a single-dose gene therapy (AMT-130) was delivered during a 12 to 18-hour brain surgery, targeting the mutated huntingtin gene via viral delivery of microRNA to block toxic protein production. The trial involved 29 patients and showed sustained effects on motor function, cognition, and overall daily life outcomes. Experts say this is a monumental step — shifting Huntington’s from the “untreatable” column into one where patients may enjoy far longer, more stable lives.