UniQure to Report Four-Year Results from Huntington's Gene Therapy Trial

Source Summary

UniQure is expected to release four-year results from its clinical trial of a gene therapy for Huntington's disease before the end of September. The readout from this closely monitored trial will provide updated efficacy and safety data for the investigational treatment.

Why it matters

The four-year readout will provide critical long-term efficacy and safety data for a gene therapy approach to Huntington's disease, a rare neurodegenerative disorder with limited treatment options.

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• STAT News ↗