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UniQure Receives FDA Meeting Minutes on AMT-130 While Community Support Remains Strong
Latest News
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Feeding the Brain Through the Gut: How Prebiotics Might Shape Huntington’s Disease
Studies in mice that model Huntington’s disease suggest that prebiotics – the food that feeds our gut bacteria – might improve gut health and neurological symptoms.
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An Old Drug, New Tricks: Sertraline May Lighten the Load in HD by Targeting Protein Production
New research suggests the antidepressant sertraline might have improved function in people with Huntington’s disease, prevented motor problems in mice, and stabilized protein production. Could this common drug be impacting HD from multiple angles?
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November 2025: This Month in Huntington’s Disease Research
From stem-cell “tooth fairy therapy” to gene therapy roadblocks and new genetic insights, November was big for HD research. Catch all of these stories in our monthly summary!
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Folding A Shirt With 12-Foot Arms: Understanding Protein Folding in Huntington’s Disease
In a recent Nature Communications publication, a team of researchers investigated a new way to improve the protein folding process in HD and stop the formation of toxic clumps.
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SOM3355 Moves Toward Phase 3 as Both EMA and FDA Signal Support
SOM3355, a drug to help manage HD symptoms, has received a positive opinion from the EMA for orphan drug designation, and the company is aligned with the FDA after its End-of-Phase-2 meeting. A global Phase 3 trial is expected to start in 2026.
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A Tiny Genetic Tweak Could Delay Symptoms and Activate Cellular Cleanup for Huntington’s Disease
Scientists discover a rare genetic quirk that may extend the age of onset in people with Huntington’s disease by boosting cellular cleanup.
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Unzipping the Secrets of DNA Repair
The structure of the MutSβ molecular machine gives us more clues to the mechanism of CAG repeat expansion and how we can target it.
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Tooth fairy therapy? Stem cells from teeth tested in a small Huntington’s disease trial
A trial tested stem cells from human teeth as a possible treatment for HD. The approach appeared safe with some improvements in symptoms, but the small size of the study and the unclear underlying science of this approach means caution is urged.
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New insights into intermediate CAG repeats: Mapping the expansion continuum
A new study uses ultra-sensitive research techniques to map DNA changes across the entire spectrum of CAG repeat lengths, revealing where intermediate repeats fit in the genetic landscape
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UniQure and FDA No Longer in Alignment on Approval Pathway for AMT-130
In a new press release, uniQure shared that the FDA no longer agrees with their approach for advancing AMT-130, specifically the use of an external control group. The path forward is uncertain, but uniQure remains committed to the HD community.
