Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Recently the Critical Path Institute announced a new effort - the Huntington's Disease Regulatory Science Consortium, or HD-RSC. This collaboration with many international partners aims to speed the development of new HD treatments. What's all this about, and how could it help HD patients?
A new analysis of clinical data from the TRACK-HD and COHORT studies proposes a way to design of clinical trials designed to delay the onset of HD, rather than treating symptoms after they occur.
If media reports of a "wonder-drug" that could "stop all neurodegenerative brain diseases, including dementia” seem too good to be true, that's because they are. The truth behind the headlines is that researchers tested thousands of already-licensed drugs in worms, and a couple that went on to show beneficial effects in mouse models of two rare forms of dementia. While it gives researchers two new leads, this research doesn't prove anything about these drugs in patients with neurodegenerative diseases, and has virtually nothing to do with Huntington's disease at all.
Significant news for the Huntington's disease community this week, as the USA's drug regulator, the FDA, has formally approved Austedo, also known as deutetrabenazine, for prescribing in HD. This modified form of tetrabenazine helps control chorea, the jerky movements often found in HD patients, but is taken twice rather than three times a day.
Recent days have seen a slew of news emerging regarding the use of something called genome editing as a potential therapy for genetic diseases like Huntington's Disease. These approaches, which include exotic sounding tools like zinc finger nucleases and CRISPR/Cas9, differ from more traditional ways reducing the impact of the HD mutation on cells. What's new in this exciting area of research?
A recent press release from Teva Pharmaceuticals has the HD community excited, claiming "Pridopidine Demonstrates Slowing of Progression of Huntington Disease in PRIDE-HD Study". What's pridopidine, and what can we really say about HD progression in patients treated with it?
Figuring out the shape of a protein can help scientists understand how it works and what goes wrong in disease. Huntingtin, the protein that causes Huntington's disease, has been an elusive target. A recent study using electron microscopes offers a striking glimpse of huntingtin, paving the way for future work.
Clumps of mutant huntingtin protein in brain cells are a hallmark of HD, and they build up slowly, occupying more and more cells over time. Recent research in mice shows that the harmful proteins can travel between neurons, setting off a chain reaction that leads to more sick cells and the development of symptoms.
A recent article in the UK newspaper the Daily Telegraph has HD families very excited. The title, "First drug to reverse Huntington’s disease begins human trials", certainly sounds exciting! But what's really going on? HDBuzz is here to help us untangle hope from hype in the huntingtin lowering world.