Sad news from Novartis: dosing suspended in VIBRANT-HD trial of branaplam

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.

Dr Leora FoxAugust 08, 2022

Updates from uniQure about their gene therapy for Huntington's disease

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.

Dr Leora Fox and Dr Rachel HardingJuly 11, 2022

Huntington’s disease therapeutics conference 2022 - Day 2

Huntington’s disease therapeutics conference 2022 - Day 2

Check out research updates from Day 2 of the 2022 HD Therapeutics Conference #HDTC2022

Dr Sarah Hernandez, Dr Leora Fox, Dr Rachel Harding, and Joel StantonJune 21, 2022

Artificial intelligence to save the day? How clever computers are helping us understand Huntington’s disease.

Artificial intelligence to save the day? How clever computers are helping us understand Huntington’s disease.

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.

Dr Rachel HardingJune 21, 2022

Huntington’s disease therapeutics conference 2022 - Day 1

Huntington’s disease therapeutics conference 2022 - Day 1

Check out research updates from Day 1 of the 2022 HD Therapeutics Conference #HDTC2022

Dr Rachel Harding, Dr Leora Fox, Dr Sarah Hernandez, and Joel StantonJune 20, 2022

A new roadmap to track Huntington’s disease progression

A new roadmap to track Huntington’s disease progression

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.

Dr Sarah HernandezJune 15, 2022

Revisiting vitamin therapy for HD

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).

Makenna May and Dr Leora FoxMay 19, 2022

A spoonful of branaplam helps the huntingtin go down

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 Huntington’s. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.

Dr Rachel HardingMarch 16, 2022

Huntington’s disease therapeutics conference 2022 - Day 3

Huntington’s disease therapeutics conference 2022 - Day 3

Check out research updates from Day 3 of the 2022 HD Therapeutics Conference #HDTC2022

Dr Sarah Hernandez, Dr Leora Fox, Dr Rachel Harding, and Joel StantonMarch 08, 2022

Shining a spotlight on huntingtin: a tool to measure huntingtin-lowering in real time

Shining a spotlight on huntingtin: a tool to measure huntingtin-lowering in real time

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

Dr Rachel HardingFebruary 17, 2022

BAC to basics: a more accurate mouse model for Huntington's disease

BAC to basics: a more accurate mouse model for Huntington's disease

A genetically-tweaked Huntington's disease mouse model shows a tendency for the CAG repeat to grow, just like we see in humans with the mutation.

Dr Michael FlowerFebruary 10, 2022