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
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?
The first round of findings from the halted tominersen huntingtin lowering trial, GENERATION-HD1, run by Roche were shared this week with the HD community. HDBuzz explains what they found and what’s next.
In a much-needed bit of good news for the Huntington's disease community, Neurocrine Bioscience's KINECT-HD trial showed that treatment with valbenazine significantly reduced the involuntary movements called chorea
New tools let us “see” clumps of toxic huntingtin protein which build up in the brains of people with Huntington’s disease over time. Tracking these clumps might help us to better understand how HD progresses or how treatments might slow or halt HD.
The HDBuzz team sat down (virtually) for an in depth Q&A session with the team at Roche to answer questions about tominersen and the recent halting of the GENERATION-HD1 trial
A new system has been developed that allows researchers to fine-tune gene expression with oral drugs, work that provides a powerful tool for gene editing.