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?
Earlier this week, Ionis Pharmaceuticals put out a press release after presenting data from the HTTRx (RG6042) Huntingtin-lowering clinical trial program at a scientific conference. Ionis’ partner Roche also released a community statement. Our friend at Huntington’s Disease Society of America, Dr Leora Fox, has prepared this Q&A in response to community questions received after the release. We liked it so much, we decided to reproduce it here, by kind permission, for the benefit of HDBuzz readers.
Drs Rachel Harding and Tam Maiuri are taking a leap of faith to make discoveries in Huntington’s Disease (HD) research swift and open to all. They are publishing their lab notes on fundamental HD research online in real time, at the risk of losing credit for their work. As a result, they hope to speed the path of discovery to more effective HD treatments.
The cause of Huntington’s disease has been known since 1993, but the physical structure of the healthy huntingtin protein proved difficult to discover until now. German scientists have now revealed the shape of the huntingtin protein for the first time. Although the structure of the mutant form of the protein was not investigated, this study provides an excellent platform to build upon and will boost drug development efforts.
Good morning from the final day of the 2018 HD Therapeutics Conference! Two sessions today, the first focused on the protein made from the HD gene. The second includes updates on Huntingtin Lowering Trials from both Wave Life Sciences and Ionis Pharmaceuticals.
Updates from day 2 of the Huntington's Disease Therapeutics Conference focusing on DNA repair in HD.
Jeff and Ed report from the Huntington's Disease Therapeutics Conference - the biggest annual gathering of HD researchers. This year's conference is bigger and more exciting than ever.
New hints are emerging about the normal role of the gene that causes Huntington's disease. A recent report uses cutting edge techniques to study this question in cells growing in the lab. We'll help separate the fascinating new science from some scary-sounding headlines.
A recent mouse study of a drug known as CTEP suggests the drug is surprisingly helpful for HD-like symptoms in mice. This is a welcome surprise, because it suggests a well-understood brain process might be a useful drug target for future HD research.