UniQure und die FDA sind sich beim Zulassungsverfahren für AMT-130 nicht mehr einig.
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 […]
Oktober 2025: Dieser Monat in der Huntington-Forschung
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 […]
Eine Karte durch die Zeit: Die Huntington-Krankheit von Geburt an im Gehirn verfolgen
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 […]
Zusätzliche Klarheit: Was wir 4 Wochen nach den uniQure-Nachrichten wissen
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, […]
Wie groß ist der Eisberg der Huntington-Krankheit?
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 […]
Kongress zur klinischen Forschung zur Huntington-Krankheit 2025 – Tag 3
Willkommen zurück zum letzten Tag des Kongresses für klinische Forschung zur Huntington-Krankheit (HK) in Nashville, Tennessee! Translationale Probleme bei der HK Die erste Sitzung konzentriert sich auf translationale Probleme bei der HK – wie wir die Forschung zu den Menschen bringen, die sie am dringendsten benötigen: den HK-Familien. Dr. Sarah Tabrizi vom UCL eröffnete die Veranstaltung mit einer Einführung, in der sie translationale Probleme diskutierte […]
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