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.
Long repetitive sequences of C-A-G letters in the DNA code are associated with at least 12 genetic diseases, including Huntington’s disease (HD). A group of scientists in Massachusetts, USA, have ...
CAG repeats can get longer over time as the HD gene is used, like the growing potholes and cracks in an old road. New ...
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 ...
We all know heart health matters – but what if the same habits that keep your heart strong could also protect your brain? A new study reveals a link between cardiovascular health and lower levels of ...
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 ...
On 25th June 2024, Wave Life Sciences shared the results of their SELECT-HD clinical trial. This trial tested a therapy called WVE-003, designed to only lower the expanded, harmful form of the ...
Imagine a vast and intricate factory, humming with activity. This factory isn’t manufacturing cars or electronics but rather the essential components that keep our bodies running. Inside each cell, ...
We’re proud to announce Gravity Guignard as a 2025 HDBuzz Prize winner! A new study points to one of the reasons why past HD trials might have failed. The good news? This shows the path to safer drugs ...
We’re back for day 2 of the CHDI Huntington’s Disease Therapeutics Conference! We’re kicking things off with some exciting talks on genetic modifiers and learning how we can advance them toward ...
This week, we heard an update from Roche about their huntingtin-lowering therapy, tominersen, currently being tested in the GENERATION HD2 trial. An independent data monitoring committee (iDMC) that ...