Key Takeaways

A high-dose of AMT-130 slowed Huntington’s disease progression by 75% over 36 months

Most side effects were related to surgery and resolved, researchers said

If approved, this would be the first genetic therapy for Huntington’s disease

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 study, patients given a high dose of UniQure’s experimental therapy AMT-130 experienced a 75% slowing of disease progression after three years, the company said. The therapy also reduced levels of a key marker of brain damage — neurofilament light protein in spinal fluid — by an average of 8.2%.

"These findings reinforce our conviction that

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