• A significant, dose-dependent, slowing in disease progression measured by cUHDRS was observed through 24 months in patients receiving the high dose of AMT-130.
• Trends in measurements of motor and cognitive function showed near-baseline stability throughout the 24 months of follow-up in patients receiving the high dose of AMT-130.
• A statistically significant reduction of NfL in cerebral spinal fluid (CSF) was observed in patients treated with AMT-130.
• Based on data observed to date, AMT-130 remains generally well-tolerated, with a manageable safety profile at both doses. There were no new AMT-130-related serious adverse events reported.
uniQure Press Release: Press Releases | Investors & Media | uniQure
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