featured
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New insights into intermediate CAG repeats: Mapping the expansion continuum
8 min read
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
8 min read
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
6 min read
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
8 min read
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
19 min read
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?
10 min read
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.
By Dr Chris Kay -
Huntington’s Disease Clinical Research Congress 2025 – Day 3
30 min read
Catch up on all the latest HD news from Day 3 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
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Huntington’s Disease Clinical Research Congress 2025 – Day 2
23 min read
Catch up on all the latest HD news from Day 2 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
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Huntington’s Disease Clinical Research Congress 2025 – Day 1
11 min read
Catch up on all the latest HD news from Day 1 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
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Controlling DNA Scanning Machines to Slow Expansion of CAG Repeats
5 min read
A new study identifies small molecules that target a DNA repair machine, opening a potential path to delay Huntington’s disease onset.