On 24 September, uniQure announced exciting topline results for their Phase 1/2 study of AMT-130 in HD. The high dose of AMT-130 met the study’s primary endpoint by demonstrating statistically significant slowing of disease progression (as assessed by the composite Unified Huntington’s Disease Rating Scale) at 36 months compared to a propensity score-matched external control (Enroll-HD natural history data). The high dose of AMT-130 also demonstrated statistically significant slowing of disease progression as measured by Total Functional Capacity, a key secondary endpoint, at 36 months compared to a propensity score-matched external control.

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