Nuevas perspectivas sobre las repeticiones CAG intermedias: mapeando el continuo de expansión
Why do some people with intermediate CAG repeats, a genetic “gray zone” in Huntington’s disease, develop neurological symptoms while others do not? This article covers a recent study that tackled this question by looking for somatic expansion, which is a tiny changes in DNA, in individuals across various CAG repeat sizes. Using ultra-sensitive research technology […]
UniQure y la FDA ya no coinciden en la vía de aprobación de AMT-130.
In a very brief press release from uniQure on November 3, 2025 we learned that the company is no longer aligned with the FDA on moving their drug AMT-130 forward. Just a few weeks ago, positive news from their ongoing clinical trials took the world by storm. Their goal was to use those data to […]
Octubre de 2025: Este mes en la investigación de la enfermedad de Huntington
This month brought new revelations across biomarkers, basic science and community gathering. In particular, we saw how DNA-repair targeting strategies are moving into focus, how gaps in diagnosis are being quantified, and how the annual Huntington’s Disease Clinical Research Congress 2025 deepened connections across the field. Together these developments reflect how far the HD research […]
Un mapa a través del tiempo: el seguimiento de la enfermedad de Huntington desde el nacimiento en el cerebro
A new study in mouse models reveals how Huntington’s disease (HD) disrupts brain development over time, even long before symptoms appear. Using advanced sequencing tools and spatial transcriptomics, a technique that maps where in the brain genes are activated, researchers discovered early warning signs that could help explain why some brain cells are more vulnerable […]
Claridad adicional: Lo que sabemos 4 semanas después de la noticia uniQure
The past 4 weeks have been a whirlwind in the Huntington’s disease (HD) community. On September 25th we had an update from uniQure about a drug they’re testing for HD in ongoing clinical trials. The news was positive and it took the world by storm, producing jaw dropping headlines from news sources around the world, […]
¿Qué tan grande es el iceberg de la enfermedad de Huntington?
Huntington’s disease (HD) is caused by repeating C-A-G letters of genetic code that are too long. Everyone who develops HD is born with 36 or more CAG repeats, but not everyone with 36 or more CAG repeats is actually diagnosed with HD. That’s because either they are not old enough yet to have symptoms, or […]
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