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Four-Year Data Dims Hope for Huntington's Drug
29 Sep
Summary
- Huntington's disease gene therapy benefits declined after four years.
- Small trial results may hinder US regulatory approval efforts.
- UniQure NV experienced a significant stock price drop post-announcement.
UniQure NV's gene therapy candidate for Huntington's disease, AMT-130, has encountered a significant hurdle. Recent four-year trial data revealed that the observed benefits of the drug have diminished, presenting a less optimistic outlook compared to earlier three-year results.
This development could complicate the company's efforts to gain approval from US regulators for the treatment. Following the announcement of these findings, UniQure NV experienced a notable decline in its stock value during early trading sessions.
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