Clinical Trial SuccessAMT-130 showed statistically significant and dose-dependent slowing of disease progression and a reduction in neurofilament light chain at 24 months.
FDA AlignmentQURE announced they have reached regulatory alignment on a path to accelerated approval with the FDA that will not require them to run an additional study, removing the overhang on the stock for many investors.
Regulatory ApprovalUniQure disclosed a positive regulatory update for AMT-130 in Huntington’s disease, with an agreement on key elements of an Accelerated Approval pathway.