Researchers with PTC Therapeutics recently published exciting new findings - a promising new huntingtin lowering drug that can be taken as a pill. Will this change how we move forward with huntingtin lowering?
The first round of findings from the halted tominersen huntingtin lowering trial, GENERATION-HD1, run by Roche were shared this week with the HD community. HDBuzz explains what they found and what’s next.
In a much-needed bit of good news for the Huntington's disease community, Neurocrine Bioscience's KINECT-HD trial showed that treatment with valbenazine significantly reduced the involuntary movements called chorea
New tools let us “see” clumps of toxic huntingtin protein which build up in the brains of people with Huntington’s disease over time. Tracking these clumps might help us to better understand how HD progresses or how treatments might slow or halt HD.
The HDBuzz team sat down (virtually) for an in depth Q&A session with the team at Roche to answer questions about tominersen and the recent halting of the GENERATION-HD1 trial
A new system has been developed that allows researchers to fine-tune gene expression with oral drugs, work that provides a powerful tool for gene editing.
Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.
A new study by researchers at Johns Hopkins describes a non-invasive way to track progression of Huntington’s disease. This could be used before patients even start showing symptoms to help test treatments in early stages of disease.
A successful early trial of a drug for Familial Transthyretic Amyloidosis showed that CRISPR gene editing could be safely used in the human body. What does this mean for gene editing in HD?