Huntington's Hopes.

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Bibliographic Details
Title: Huntington's Hopes.
Authors: Parshall, Allison (AUTHOR)
Source: Scientific American. Jan2026, Vol. 334 Issue 1, p8-11. 4p. 1 Color Photograph.
Subjects: Huntington disease, Gene therapy, Clinical trials, Disease progression, Brain surgery, Health services accessibility, Drug approval
Abstract: The article discusses the promising results of AMT-130, an experimental gene therapy for Huntington's disease, which appears to slow disease progression. Conducted by uniQure, the phase ½ clinical trials involved 24 participants and indicated that a high dose of AMT-130 reduced the rate of progression by 75% compared to an external control group. The treatment requires invasive brain surgery to deliver the drug directly into affected neurons and aims for FDA accelerated approval by the end of 2026. While this development offers hope to the Huntington's community, concerns remain regarding the treatment's cost and accessibility. [Extracted from the article]
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Database: Psychology and Behavioral Sciences Collection
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  Data: Huntington's Hopes.
– Name: Author
  Label: Authors
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  Data: <searchLink fieldCode="AR" term="%22Parshall%2C+Allison%22">Parshall, Allison</searchLink> (AUTHOR)
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  Data: <searchLink fieldCode="JN" term="%22Scientific+American%22">Scientific American</searchLink>. Jan2026, Vol. 334 Issue 1, p8-11. 4p. 1 Color Photograph.
– Name: Subject
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  Data: <searchLink fieldCode="DE" term="%22Huntington+disease%22">Huntington disease</searchLink><br /><searchLink fieldCode="DE" term="%22Gene+therapy%22">Gene therapy</searchLink><br /><searchLink fieldCode="DE" term="%22Clinical+trials%22">Clinical trials</searchLink><br /><searchLink fieldCode="DE" term="%22Disease+progression%22">Disease progression</searchLink><br /><searchLink fieldCode="DE" term="%22Brain+surgery%22">Brain surgery</searchLink><br /><searchLink fieldCode="DE" term="%22Health+services+accessibility%22">Health services accessibility</searchLink><br /><searchLink fieldCode="DE" term="%22Drug+approval%22">Drug approval</searchLink>
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  Label: Abstract
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  Data: The article discusses the promising results of AMT-130, an experimental gene therapy for Huntington's disease, which appears to slow disease progression. Conducted by uniQure, the phase ½ clinical trials involved 24 participants and indicated that a high dose of AMT-130 reduced the rate of progression by 75% compared to an external control group. The treatment requires invasive brain surgery to deliver the drug directly into affected neurons and aims for FDA accelerated approval by the end of 2026. While this development offers hope to the Huntington's community, concerns remain regarding the treatment's cost and accessibility. [Extracted from the article]
– Name: AbstractSuppliedCopyright
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  Group: Ab
  Data: <i>Copyright of Scientific American is the property of Scientific American and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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    Identifiers:
      – Type: doi
        Value: 10.1038/scientificamerican012026-5rsmeajbz76o5mligkrnvl
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      – Code: eng
        Text: English
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      Pagination:
        PageCount: 4
        StartPage: 8
    Subjects:
      – SubjectFull: Huntington disease
        Type: general
      – SubjectFull: Gene therapy
        Type: general
      – SubjectFull: Clinical trials
        Type: general
      – SubjectFull: Disease progression
        Type: general
      – SubjectFull: Brain surgery
        Type: general
      – SubjectFull: Health services accessibility
        Type: general
      – SubjectFull: Drug approval
        Type: general
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      – TitleFull: Huntington's Hopes.
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              Text: Jan2026
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              Y: 2026
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