clinical-trial
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uniQure gets the green light to resume testing HD gene therapy
4 min read
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
5 min read
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
11 min read
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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Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials
8 min read
After receiving a high dose of uniQure’s gene therapy for Huntington’s disease, a few patients experienced serious side effects, but are now recovering. HDBuzz explores what this means for the experimental huntingtin-lowering drug, AMT-130.
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Sad news from Novartis: dosing suspended in VIBRANT-HD trial of branaplam
7 min read
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 -
Updates from uniQure about their gene therapy for Huntington's disease
10 min read
uniQure is conducting safety trials of the first gene therapy for HD. A recent press release provided a 1 year update on the first group of 10 people who underwent surgery to receive this experimental drug. Let’s talk more about what it means.
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Artificial intelligence to save the day? How clever computers are helping us understand Huntington’s disease.
5 min read
Scientists at IBM and the CHDI Foundation have used artificial intelligence to analyse datasets from Huntington’s disease observational trials to model progression of the disease. They hope their findings will help improve clinical trial design.
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A new roadmap to track Huntington’s disease progression
7 min read
Researchers have updated the system that classifies Huntington’s disease progression. Tracking progression in 4 stages will make clinical trial screening and data interpretation easier and faster, and pave the way for pre-symptomatic trials.
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Revisiting vitamin therapy for HD
6 min read
A small clinical trial of high-dose biotin and thiamine for treating HD is being planned in Spain. This trial is based on research that links HD to another neurodegenerative disease called biotin-thiamine responsive basal ganglia disease (BTBGD).
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A spoonful of branaplam helps the huntingtin go down
7 min read
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.