Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
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.
The research news, announced on 11 December 2017, that a research team from the Huntington's Disease Centre at University College London have made significant step towards a possible treatment for Huntington's disease, raised many questions for the Huntington's disease community. Dr Ed Wild answered some of these questions on behalf of the UK HD Association, helping to give some context to what this news means for people affected by Huntington's now and in the future.
In an announcement likely to stand as one of the biggest breakthroughs in Huntington's disease since the discovery of the HD gene in 1993, Ionis and Roche today announced that the first human trial of a huntingtin-lowering drug, IONIS-HTTRx, demonstrates that it reduces mutant huntingtin in the nervous system, and is safe and well-tolerated.
Genome editing is a much-discussed frontier in medical science at the moment, with ‘DNA surgery’ having the potential to treat or cure genetic diseases like Huntington’s. Here we look at what this technology can currently do and discuss the challenges that still lie in the way. We’ll also discuss how a team of Swiss scientists have recently developed a way to switch off the genome-editing machinery after it’s done its job.
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.