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What to Expect From UniQure's Upcoming Huntington's Gene Therapy Readout
Key Takeaways
- UniQure is expected to report four-year results from its closely tracked gene therapy trial in Huntington's disease before the end of September.
- The readout is significant because Huntington's has no disease-modifying treatment and gene therapy has yet to produce a clear win in neurodegeneration.
- Key items to watch include long-term safety, durability of effect, biomarkers such as mutant huntingtin and neurofilament light, and clinical rating scales.
- The data will shape next steps for the program and inform how the field views direct-delivery gene therapies for the central nervous system.
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DT Editorial Team··via statnews.com