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Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
Latest News
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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.
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Huntington’s Disease Clinical Research Congress 2025 – Day 3
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
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
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
A new study identifies small molecules that target a DNA repair machine, opening a potential path to delay Huntington’s disease onset.
HDBuzz Trial Tracker
Sites for Phase 3 trial for votoplam, INVEST-HD, now open. Updates on recruitment and first dosing imminent.
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
