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What will be the efficacy of uniQure's AMT-130 therapy in Phase III trials by June 30, 2025?
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uniQure official press releases and scientific publications
Story
uniQure Stock Soars 27% on Positive Interim Data for AMT-130 Huntington’s Disease Therapy
Jul 9, 2024, 12:27 PM
uniQure has announced positive interim data from its Phase I/II trials of AMT-130, a one-time therapy for Huntington’s disease. The data demonstrated a statistically significant, dose-dependent, and durable evidence of therapeutic benefit, with patients receiving high-dose AMT-130 showing an 80% slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale (cUHDRS). Significant improvements were also observed in neurofilament light chain (NFL) levels. The announcement has led to a 27% increase in uniQure's stock price. Despite the low numbers required given the risk of the intervention, this development marks a significant step towards the first disease-modifying treatment for Huntington’s disease.
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