Huntington’s disease is a neurological (brain) condition that causes involuntary movement, changes to emotions, behavior, and thinking. Huntington’s is a genetic disease that worsens over time.

An experimental gene therapy slowed Huntington’s by up to 75 percent in a small clinical trial. While not a cure, it may give patients longer lives.

Huntington's disease findings were announced last month in a news release from uniQure, the company that sponsored the research.

Huntington’s disease has been treated for the first time. The genetic disease has no cure or currently approved treatments, but the company uniQure developed and tested a drug, known as AMT-130, on 29 ...

After years of heartbreak, researchers have found an experimental treatment that can slow the progression of Huntington’s ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

As well as slowing disease progression by 75 percent at 36 months, the scientists found that neurofilament light protein (NfL)—a biomarker of neuronal damage—levels in spinal fluid were lower in ...

FRIDAY, Sept. 26, 2025 (HealthDay News) — A new gene therapy has shown promise in slowing the progression of Huntington’s disease, according to early trial results released Wednesday. In a Phase 1/2 ...