huntingtin-lowering
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Steady progress from uniQure – promising data to end the year
uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials
By Dr Leora Fox -
Could halting CAG expansions be a new treatment for HD?
The gene MSH3 is getting a lot of attention in HD research lately. New findings suggest MSH3 lowering could halt CAG repeat expansions, offering a new therapeutic avenue.
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Updates from PTC Therapeutics and uniQure on their huntingtin-lowering trials
Both PTC Therapeutics and uniQure have shared updates from their respective clinical trials, testing different huntingtin-lowering approaches. We explore the data presented from both of these studies and what this means for HD family members.
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Hunting for balance: how the huntingtin protein compensates in HD
Researchers look at the cause and effect of various forms of the HTT protein. They find both expanded and unexpanded HTT contribute to brain cell communication and the brain has an amazing capacity to compensate for changes related to disease
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Roche Phase II GENERATION HD2 study underway
Roche released a community letter in early 2023, to share that their Phase II clinical trial to study the huntingtin-lowering drug, tominersen, is now underway. In this article, we summarise the latest news about this huntingtin-lowering drug.
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uniQure gets the green light to resume testing HD gene therapy
Following a 3-month pause in enrollment due to concerns about side effects, uniQure shared the good news that their trial of the HD gene therapy AMT-130 will continue as planned, with new safety measures in place.
By Dr Leora Fox -
Update on the PTC Therapeutics PIVOT-HD Trial
Last week, PTC Therapeutics released a statement sharing that recruitment of participants into the US arm of the PIVOT-HD trial has been paused. In this article, we will lay out exactly what is known and what this announcement means.
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Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials
Two companies developing medicines for Huntington’s disease announced news about their huntingtin-lowering drugs. Let’s talk about the history and the caveats surrounding these hopeful pieces of news.
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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