clinical-trial
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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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PROOF-HD study of pridopidine ends with negative result
The phase 3 trial missed its primary endpoint of slowing loss of function in Huntington’s 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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Disappointing news from Novartis about branaplam and the VIBRANT-HD trial
Novartis have announced that they are ending development of the drug branaplam in Huntington’s disease. Here, we review this latest news and its impact on the HD community.
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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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Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials
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
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
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.