uniQure says AMT-130 Phase I/II data show 80% slowing on cUHDRS at 36 months in high-dose Huntington’s patients

PUBT · 2d ago
uniQure says AMT-130 Phase I/II data show 80% slowing on cUHDRS at 36 months in high-dose Huntington’s patients
  • uniQure released new follow-up data from ongoing Phase I/II studies of AMT-130 in Huntington’s disease, extending results through 48 months.
  • The update showed continued slowing of disease progression in high-dose patients versus an external natural-history comparator, with functional benefit maintained over four years.
  • Results also suggested a dose-dependent effect, with high-dose patients performing better than low-dose patients on overall disease and daily-function measures.
  • The data were not included in the already submitted accelerated-approval application, with the company indicating the findings will be presented at a future scientific meeting.
  • Safety remained broadly manageable, with earlier treatment-related central nervous system inflammation events reported as resolved.


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. uniQure NV published the original content used to generate this news brief via GlobeNewswire (Ref. ID: 202609290712PRIMZONEFULLFEED9835905) on September 29, 2026, and is solely responsible for the information contained therein.