Tag: AMT-130
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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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Promising Huntington’s disease gene therapy slows progression, but questions remain
What the early results show News from a European biotech company on a new gene therapy has sparked guarded optimism in the Huntington’s disease community. The approach, delivered during brain surgery, targets the genetic error that drives the condition. In a small, preliminary trial involving roughly 30 participants, the highest dose of the treatment appeared…
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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…