SOM3355 Moves Toward Phase 3 as Both EMA and FDA Signal Support
SOM3355 is an investigational therapy aimed at managing multiple symptoms of Huntington’s disease (HD) and recently crossed two key regulatory milestones. In September, the European Medicines Agency (EMA) issued a positive opinion supporting orphan drug designation for SOM3355. Now, following a productive End-of-Phase-2 meeting in the United States, the US Food and Drug Administration (FDA) […]
A Tiny Genetic Tweak Could Delay Symptoms and Activate Cellular Cleanup for Huntington’s Disease
Scientists often use genetics, the study of DNA, to understand the cellular changes that cause disease. By comparing people’s DNA with their symptoms, they can pinpoint specific genetic differences, called variants, that influence the severity of a disease. Huntington’s disease (HD) is well-suited for genetic analysis because of its well-understood genetic roots – an expansion […]
Unzipping the Secrets of DNA Repair
Our bodies are experts at looking after our DNA and are continuously monitoring for any damage that needs to be repaired to keep us healthy. Parts of DNA that are very repetitive, like the sequence causing Huntington’s disease (HD), are very tricky to look after and our body can try and fix them but make […]
Tooth fairy therapy? Stem cells from teeth tested in a small Huntington’s disease trial
A small study from Brazil tested whether stem cells from human dental pulp, the soft tissue inside teeth, could help people with Huntington’s disease (HD). The results hint at small improvements on some movement measures, but the study had few participants who were tracked over a short timeframe, and many questions remain that raise red […]
New insights into intermediate CAG repeats: Mapping the expansion continuum
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 and FDA No Longer in Alignment on Approval Pathway for 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 […]
EN 
ES 
IT 
DE 
FR 