Tag: uniQure
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Huntington’s disease gene therapy offers hopeful prospects, questions
Promising but preliminary: what the results suggest A potential breakthrough in Huntington’s disease treatment has sparked cautious optimism within the community. The European biotech firm uniQure reported top-line findings from a small, early-stage trial of AMT-130, a gene therapy designed to address the genetic error behind Huntington’s disease. According to the company, those receiving the…
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Huntington’s Disease Gene Therapy: Promise Amid Unanswered Questions
New Hope in Huntington’s Disease Gene Therapy A European biotech company, uniQure, has released preliminary results from an experimental gene therapy (AMT-130) aimed at Huntington’s disease. The data suggest the therapy could slow disease progression by about 75% over three years in patients receiving the highest dose. The treatment is delivered directly into the brain…
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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…