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Folding A Shirt With 12-Foot Arms: Understanding Protein Folding in Huntington’s Disease
Latest News
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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.Â
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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.
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October 2025: This Month in Huntington’s Disease Research
October was an insight-rich month in Huntington’s disease research: additional clarity around uniQure’s AMT-130, mapping how and where HD changes the brain, early mechanistic studies targeting DNA repair, and a major meeting gathering the field.
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A Map Through Time: Tracking Huntington’s Disease From Birth in the Brain
Why do some brain cells die first in HD? New study shows changes in gene activity begin at birth in mice, especially in striatum & cortex—mapped with spatial & single-cell tools.
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Additional Clarity: What We Know 4 Weeks After the uniQure News
We’re 4 weeks out from uniQure’s positive update about AMT-130 that took the world by storm. Does the data match the media hype? Where do uncertainties remain? And what are some of the key questions from the Huntington’s disease community?
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How Big Is The Huntington’s Disease Iceberg?
We can count how many people are seen in a clinic with Huntington’s disease by genetic testing, but how many people with the gene for HD are never diagnosed by a doctor? A new mathematical approach seeks to revisit this tricky question.