UniQure reports 80% slowing of Huntington's disease progression in Phase I/II trials of AMT-130.

UniQure reports 80% slowing of Huntington's disease progression in Phase I/II trials of AMT-130, with 24 months of follow-up data from 29 treated patients. High-dose patients experienced this slowing compared to a propensity score-weighted external control. UniQure plans to hold an RMAT meeting with the FDA for expedited clinical development.