A new hope for patients with Huntington's disease?

On Sept 24, a gene therapy company uniQure released preliminary positive data from its pivotal phase 1/2 trial of AMT-130, a novel treatment for Huntington's disease. The announcement sparked widespread discussion in the medical community and renewed hope among patients with Huntington's disease and clinicians. Huntington's disease so far has been thought to be an incurable disease with little recent progress in the development of new treatment modalities.

Authors

Publication Details

Journal
EClinicalMedicine
Published
2025-10-01
DOI
https://doi.org/10.1016/j.eclinm.2025.103612
Citations
2
Primary Topic
Genetic Neurodegenerative Diseases
Type
article
Field-Weighted Citation Impact
1.82
Controls
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article

A new hope for patients with Huntington's disease?

eClinicalMedicine
2 citations
EClinicalMedicine
Genetic Neurodegenerative Diseases
1.82
article

A new hope for patients with Huntington's disease?

eClinicalMedicine
article en
2 citations

Abstract

On Sept 24, a gene therapy company uniQure released preliminary positive data from its pivotal phase 1/2 trial of AMT-130, a novel treatment for Huntington's disease. The announcement sparked widespread discussion in the medical community and renewed hope among patients with Huntington's disease and clinicians. Huntington's disease so far has been thought to be an incurable disease with little recent progress in the development of new treatment modalities.

EClinicalMedicineVol. 88
Openalex Percentile: Top 13%
Genetic Neurodegenerative Diseases
1.82
DOIOpen Access PDFOpenAlex
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A new hope for patients with Huntington's disease? — eClinicalMedicine · EClinicalMedicine (2025) | TGRS Research Map | TGRS