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Interim data from the ongoing HD Gene TRX-1 and 2 clinical trials.



• 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.



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