Huntington's Hopes.
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| 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] |
| 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. (Copyright applies to all Abstracts.) | |
| Database: | Psychology and Behavioral Sciences Collection |
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| FullText | Text: Availability: 1 |
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| Header | DbId: pbh DbLabel: Psychology and Behavioral Sciences Collection An: 189782883 AccessLevel: 6 PubType: Periodical PubTypeId: serialPeriodical PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Huntington's Hopes. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AR" term="%22Parshall%2C+Allison%22">Parshall, Allison</searchLink> (AUTHOR) – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22Scientific+American%22">Scientific American</searchLink>. Jan2026, Vol. 334 Issue 1, p8-11. 4p. 1 Color Photograph. – Name: Subject Label: Subjects Group: Su 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> – Name: Abstract Label: Abstract Group: Ab 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 Label: 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.) |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=pbh&AN=189782883 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1038/scientificamerican012026-5rsmeajbz76o5mligkrnvl Languages: – Code: eng Text: English PhysicalDescription: 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 Titles: – TitleFull: Huntington's Hopes. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Parshall, Allison IsPartOfRelationships: – BibEntity: Dates: – D: 01 M: 01 Text: Jan2026 Type: published Y: 2026 Identifiers: – Type: issn-print Value: 00368733 Numbering: – Type: volume Value: 334 – Type: issue Value: 1 Titles: – TitleFull: Scientific American Type: main |
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