Huntington's Hopes.
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| Title: | Huntington's Hopes. |
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| 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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| 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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| ISSN: | 00368733 |
| DOI: | 10.1038/scientificamerican012026-5rsmeajbz76o5mligkrnvl |
