Overview

• Topline data released this week report roughly 75% slower clinical progression over about 36 months in high‑dose AMT‑130 recipients versus an external matched control group.
• Coverage cites differing high‑dose sample sizes, with Newsweek and Medical Xpress noting 12 patients and Vice reporting 17, while 29 participants completed follow‑up up to 36 months overall.
• Cerebrospinal‑fluid neurofilament light, a marker of neuronal injury, declined from baseline at 36 months in treated patients.
• Investigators describe the therapy as well tolerated in this study, though it requires a lengthy MRI‑guided neurosurgical infusion into the striatum.
• The findings come from a small, nonrandomized trial using external controls and await peer review and larger controlled studies as uniQure prepares an accelerated‑approval submission in early 2026.