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Breakthrough Therapy Cuts Huntington's Progression by 75%

9/26/2025

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Summary

Story summary

UniQure's AMT-130 trial shows a 75% reduction in Huntington's progression over three years.
The therapy requires a lengthy neurosurgical procedure to deliver a modified virus to brain cells.
Patients reported improved cognitive and motor skills; some avoided wheelchairs and one returned to work.
The trial enrolled 29 participants; effects appeared at the highest dose, with FDA action planned next year.

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Story summary

UniQure's AMT-130 trial shows a 75% reduction in Huntington's progression over three years.
The therapy requires a lengthy neurosurgical procedure to deliver a modified virus to brain cells.
Patients reported improved cognitive and motor skills; some avoided wheelchairs and one returned to work.
The trial enrolled 29 participants; effects appeared at the highest dose, with FDA action planned next year.