Story perspectives
Breakthrough Therapy Slows Huntington's Disease Progression by 75%
10/3/2025
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Story summary
- AMT-130 slowed Huntington’s disease progression by 75% in 24 participants.
- It requires brain surgery lasting 8–10 hours and is not FDA-approved.
- Huntington’s disease results from an HTT gene mutation and typically begins at 30–50.
- Experts caution costs and access may be limited even if approved.
- FDA may grant accelerated approval by end of 2026; Huntington’s therapies are in development.
