Articles with the topic: clinical-trial

Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials

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.

Dr Jeff Carroll and Dr Leora FoxSeptember 30, 2022

Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials

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.

Dr Sarah Hernandez and Dr Rachel HardingAugust 29, 2022

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

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

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

Oral drug may change the story for huntingtin lowering

Oral drug may change the story for huntingtin lowering

Researchers with PTC Therapeutics recently published exciting new findings - a promising new huntingtin lowering drug that can be taken as a pill. Will this change how we move forward with huntingtin lowering?

Dr Sarah Hernandez and Dr Jeff CarrollFebruary 01, 2022