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Huntington’s disease therapeutics conference 2022 – Day 2
Check out research updates from Day 2 of the 2022 HD Therapeutics Conference #HDTC2022
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Shining a spotlight on huntingtin: a tool to measure huntingtin-lowering in real time
A new imaging tool means that scientists can now directly measure the levels of the toxic huntingtin protein in animal models of Huntington’s disease, letting us see how well huntingtin lowering therapies are working in their brains
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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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“Seeing” the toxic huntingtin protein in people with HD
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.
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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.
By Dr Leora Fox -
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.