Tag: Huntington’s Disease
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Positive Results from Gene Therapy Trial for Huntington’s Disease
Introduction to Huntington’s Disease and Gene Therapy Huntington’s disease (HD) is a debilitating, inherited neurodegenerative disorder affecting approximately 75,000 individuals across the United States, Europe, and the United Kingdom. Characterized by progressive physical and mental deterioration, this condition arises from a mutation in the huntingtin gene, which results in the accumulation of toxic proteins in…
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Breakthrough Results in Gene Therapy for Huntington’s Disease
Groundbreaking Findings from uniQure’s Gene Therapy Trial uniQure N.V. has recently announced significant results from its Phase I/II clinical trial of AMT-130, a pioneering gene therapy aimed at treating Huntington’s disease. This pivotal study reveals a remarkable 75% slowdown in disease progression among patients treated with the high dose of AMT-130 over a 36-month period…
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Positive Gene Therapy Breakthrough for Huntington’s Disease
Introduction to Huntington’s Disease and Gene Therapy Huntington’s disease is a rare yet devastating neurodegenerative disorder affecting around 75,000 individuals across the US, EU, and UK. Characterized by progressive physical and mental decline, it stems from a mutation in the huntingtin gene, which leads to the accumulation of toxic proteins in the brain. Currently, there…