Scientists engineered stem cells with “interrupted” CAG repeats to break up the toxic stretch. This may stop expansion, and could improve problems in cells that model Huntington’s disease. This study ...

POINT-HD has begun dosing its first participants with the drug RG6496, marking an early but important step for a new selective huntingtin-lowering approach.

When it comes to thinking about the effects of Huntington’s disease (HD), most people automatically start to think about the brain due to the severe symptoms caused by the breakdown of brain cells. It ...

Imagine a busy clothing factory. Proteins are like newly sewn shirts coming off the machines. They’re floppy, wrinkled, and can create a giant mess if they’re left unfolded. Normally, the cellular ...

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 ...

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, ...

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 ...

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 ...

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 ...

A BLA, or Biologics Licensing Application, is the formal dossier of information that a company uses to request permission to market a biologic, which is a therapy created from living organisms, like ...

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 ...

We’re back for Day 2 of Huntington’s Disease (HD) Clinical Research Congress in Nashville, Tennessee! Daniel Claassen from HSG and Cristina Sampaio from CHDI kick off the meeting with an overview of ...