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.
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.
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.
Check out research updates from Day 2 of the 2022 HD Therapeutics Conference #HDTC2022
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.
Check out research updates from Day 1 of the 2022 HD Therapeutics Conference #HDTC2022
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.
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).
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.
Check out research updates from Day 3 of the 2022 HD Therapeutics Conference #HDTC2022
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