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Full Steam Ahead: uniQureās On Track With Hope for Accelerated Approval of Huntingtonās Disease Drug
Latest News
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The 2025 HDBuzz Prize for Young Science Writers Is Open!
Announcing the 2025 HDBuzz Prize for Young Science Writers – sponsored by the Hereditary Disease Foundation!
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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 uniQureās gene therapy for Huntingtonās 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.
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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. Letās talk more about what it means.
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Artificial intelligence to save the day? How clever computers are helping us understand Huntingtonās disease.
Scientists at IBM and the CHDI Foundation have used artificial intelligence to analyse datasets from Huntingtonās 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 Huntingtonās disease progression
Researchers have updated the system that classifies Huntingtonās 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
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
Branaplam was originally designed to treat spinal muscular atrophy, but a new paper outlines how it could hold promise for treating Huntingtonās. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.
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Huntingtonās disease therapeutics conference 2022 – Day 3
Check out research updates from Day 3 of the 2022 HD Therapeutics Conference #HDTC2022
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Huntingtonās disease therapeutics conference 2022 – Day 1
Check out research updates from Day 1 of the 2022 HD Therapeutics Conference #HDTC2022
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Huntingtonās disease therapeutics conference 2022 – Day 2
Check out research updates from Day 2 of the 2022 HD Therapeutics Conference #HDTC2022