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Gene Therapy AMT-130 Could Slash Huntington's Symptoms by 75%

10/7/2025

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Summary

Story summary

AMT-130 gene therapy shows potential to slow Huntington's disease symptom progression by 75% over three years.
A single brain surgery delivers genetic material via a virus to reduce toxic protein production.
In a 29-patient trial, benefits were significant, though there was no placebo.
UniQure plans FDA approval in early 2026, with trials to optimize dosing and assess risks.

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Story summary

AMT-130 gene therapy shows potential to slow Huntington's disease symptom progression by 75% over three years.
A single brain surgery delivers genetic material via a virus to reduce toxic protein production.
In a 29-patient trial, benefits were significant, though there was no placebo.
UniQure plans FDA approval in early 2026, with trials to optimize dosing and assess risks.