AMT-130 Huntington’s Disease Gene Therapy: Clinical Study Outcomes at Three Years (PL6.001)

Victor Sung; Grzegorz Witkowski; Erin Furr-Stimming; Ali Samii; Christopher Ross; Bradley Elder; Ralf Reilmann; Jeffrey Long; Vicki Wheelock; Claudia Testa; Samuel Frank; Kenechi Ejebe; Wenfei Zhang; Walid Abi-Saab; David Margolin

doi:10.1212/wnl.0000000000216980

June 9, 2026Neurology

AMT-130 Huntington’s Disease Gene Therapy: Clinical Study Outcomes at Three Years (PL6.001)

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Key Points

This research aims to evaluate the effectiveness of AMT-130 gene therapy on clinical progression in participants with Huntington's disease over three years.
Evaluated participants with Huntington's disease and compared outcomes to a natural history control cohort.
Clinical progression was monitored over a three-year period.
AMT-130 demonstrated a statistically significant effect on clinical progression compared to the control cohort.
Exact metrics and specific improvements in progression rates were documented and analyzed.

Implication

Evaluates AMT-130 for clinical progression in Huntington's disease, suggesting potential benefits.

Abstract

To evaluate the effectiveness of AMT-130 on 3-year clinical progression in participants with Huntington’s disease (HD), relative to a natural history (NatHx) control cohort.

Discussion

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Cite This Study

Sung et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf3d6faef96ed7f0564ff https://doi.org/10.1212/wnl.0000000000216980

View at Publisher

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AMT-130 Huntington’s Disease Gene Therapy: Clinical Study Outcomes at Three Years (PL6.001)

Key Points

Implication

Abstract

Discussion

Cite This Study

AMT-130 Huntington’s Disease Gene Therapy: Clinical Study Outcomes at Three Years (PL6.001)

Key Points

Implication

Abstract

Discussion

Cite This Study

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