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A groundbreaking gene therapy, AMT-130, has shown remarkable promise in slowing Huntington's disease progression. Preliminary trial results from University College London indicate a 75% reduction in disease progression over three years in high-dose patients. While still in early stages, with FDA approval anticipated no earlier than 2026, this one-time treatment offers hope by targeting the disease's root cause rather than just managing symptoms. The therapy uses a harmless virus to deliver genetic instructions to the brain, silencing the harmful protein responsible for brain cell damage. Further large-scale studies are planned before widespread availability.

Prepared by Olivia Bennett and reviewed by editorial team.