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.
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.
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?
Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.
A successful early trial of a drug for Familial Transthyretic Amyloidosis showed that CRISPR gene editing could be safely used in the human body. What does this mean for gene editing in HD?
Catch up on all the latest Huntington's disease clinical trial news in this one stop shop article covering all of the recent developments in making medicines for Huntington's disease
The first group of 10 participants have been dosed in uniQure’s clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.
Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early
A gene called MSH3 helps to repair our DNA, but in HD it can slip up and cause CAG repeats to lengthen. Researchers have uncovered new information about how MSH3 activity is controlled, opening the door to new therapeutic avenues.