huntingtin-lowering
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Hereditary Disease Foundation (HDF) conference 2022 – Day 1
Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022
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Sad news from Novartis: dosing suspended in VIBRANT-HD trial of branaplam
HDBuzz is saddened to share the news that the VIBRANT-HD trial of the oral huntingtin-lowering drug, branaplam, has been temporarily suspended for the safety of participants.
By Dr Leora Fox -
A spoonful of branaplam helps the huntingtin go down
Branaplam was originally designed to treat spinal muscular atrophy, but a new paper outlines how it could hold promise for treating Huntington’s. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.
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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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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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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.