This trial tested the oral drug, branaplam, which lowered HTT but had serious safety problems, ultimately halting the trial. The results of a clinical trial, called VIBRANT-HD, investigating an oral ...

For individuals in the HD “grey zone” between 36-39 repeats, an understanding of when symptoms may arise and what kind of symptoms is critical. A new study sheds light on different sequence patterns ...

9 min read | January 2026 HD research recap: SKY-0515 shows promising safety data, community advocacy helps secure critical ...

Skyhawk Therapeutics has announced results from a Phase 1 clinical study of its experimental huntingtin-lowering drug, SKY-0515. As with all Phase 1 studies, the main goal was to understand safety and ...

Huntington’s disease (HD) is a fatal brain disorder caused by a defect in the huntingtin (HTT) gene, which produces an extra-long protein. This defect causes the HTT protein to form harmful clumps, or ...

INGREZZA is the trade name of valbenazine, a drug developed by the company Neurocrine Biosciences. It works similarly to tetrabenazine and deutetrabenazine (Austedo), drugs commonly prescribed to help ...

On September 17, 2025, we received an encouraging update from Skyhawk Therapeutics’ Phase 1 study, taking place in Australia. The recent update suggests that SKY-0515 can lower the huntingtin protein ...

Ray completed his Bachelor’s degree at the University of Toronto, Victoria College, and continued on to his graduate studies in the Department of Medical Genetics and Microbiology at the C.H. Best ...

A group of researchers at the University of Pennsylvania, USA, has recently carried out an investigation to explore whether using a type of talking therapy, called Acceptance and Commitment Therapy ...

UniQure has announced positive top-line results from its Phase I/II trial of AMT-130, a one-time gene therapy being tested in people with Huntington’s disease (HD). Topline data is a summary of the ...