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Pioneering gene therapy slows Huntington's disease
The Independent
|September 25, 2025
Scientists have slowed the progression of Huntington’s disease for the first time with a “groundbreaking” new treatment.
Experts from University College London (UCL) said the finding could “change everything” for patients with the condition, which gets worse over time and has no cure.
The disease affects movement, thinking and mood.
The study tested a new gene therapy, AMT-130, which is delivered by brain surgery.
Early-stage clinical trials involving 29 patients showed that those who were given a high dose of the treatment experienced 75 per cent less disease progression after 36 months, according to uniQure, a gene therapy company based in the Netherlands and the US.
A single dose is expected to last for a patient’s lifetime.
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