An experimental gene therapy from Uniqure slowed the progression of Huntington’s disease by 75% after three years — study results reported Wednesday that are likely to support the first approval of a genetic treatment for the rare neurodegenerative condition.
For people living with Huntington’s, an effective, one-time therapy that significantly slows the loss of muscle control and cognition around mid-life could preserve years of quality relationships and gainful employment that would normally be lost to the disease.
In a three-year analysis of the study, Huntington’s patients treated with a high dose of Uniqure’s gene therapy, called AMT-130, lost an average of 0.38 points on a measure of disease progression called cUHDRS. That compared to a loss of 1.52 points for matched participants in an external control group based on a large, natural history study. The AMT-130 benefit was statistically significant and achieved the primary goal of the study.
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