Groundbreaking Gene Therapy Holds Promise for Huntington's Disease
Gene therapy AMT-130, developed by biotech firm uniQure, shows promise in slowing Huntington's disease progression by 75%. Conducted on 29 patients, the trial highlights significant slowing of the disorder through a surgery administered once in the brain. Sarah Tabrizi calls results the most convincing in the field yet.
- Country:
- India
A pioneering gene therapy developed by biotechnology firm uniQure has demonstrated significant potential in arresting the progression of Huntington's disease by 75%, according to the latest clinical trials. The therapy, known as AMT-130, targets the huntingtin gene responsible for this inherited neurodegenerative disorder.
In the trials, 29 patients underwent the surgical procedure administering the gene therapy directly into the brain. Over a period of 36 months, researchers observed a promising reduction in disease progression, using metrics like the Unified Huntington's Disease Rating Scale and Total Functional Capacity.
Sarah Tabrizi, who spearheaded the UK trials, emphasized the groundbreaking nature of these findings, noting the urgent need for disease-modifying treatments for Huntington's. The data, yet to be peer-reviewed, signal a new beacon of hope for those affected by this debilitating condition.
(With inputs from agencies.)
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