STAT+: What to expect from UniQure’s upcoming Huntington’s gene therapy readout 

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Before the end of September, UniQure is expected to report four-year results from the closely tracked clinical trial of AMT-130, its gene therapy for Huntington’s disease.

Three-year results were reported almost one year ago. The high dose of AMT-130, administered to 12 patients, slowed the progression of Huntington’s by 75% compared to matched participants in an external control group based on a large, natural history study.

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