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Break Up With Your CAGs: How Three Letters Could Change Huntington’s Disease
Latest News
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First participants dosed in new POINT-HD huntingtin-lowering trial
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.
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BAC to basics: a more accurate mouse model for Huntington's disease
A genetically-tweaked Huntington's disease mouse model shows a tendency for the CAG repeat to grow, just like we see in humans with the mutation.
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Oral drug may change the story for huntingtin lowering
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?
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Finding the silver lining: an update on the Roche GENERATION-HD1 trial data
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.
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KINECT-HD trial shows valbenazine improves involuntary movements in Huntington's disease
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
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Updates from the EHDN Meeting 2021
Last month, HDBuzz attended the online European Huntington's Disease Network (EHDN) meeting. Read our summary of all the latest clinical trial updates.
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Real talk: Q&A with Roche about GENERATION-HD1
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
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Another tool in the box: Creation of a molecular “dimmer switch” advances gene editing
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.
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Unpacking recent gene therapy press
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.
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Does blood hold the key to testing treatments earlier in HD patients?
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.
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A first for CRISPR gene editing could have wider applications for human 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?
