Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What about this news has us so excited?
When patients participate in clinical trials, there needs to be some type of readout to determine whether the new treatment worked. It’s important to know two key things: What to measure and how to measure it. In the case of HD, these obstacles have vexed scientists and doctors for years. The latest research comes up with a clever new approach to overcome both challenges in a new way. These results could offer a valuable tool to study new HD therapeutics entering clinical trials.
Exciting technologies such as gene silencing are being developed for the treatment of Huntington’s disease. Aside from waiting for disease progression to take place, how will we know whether they are working? This has been a major hurdle for HD researchers, but we now have a super-sensitive method to measure the build-up of harmful huntingtin protein in the nervous systems of HD patients.
Our second update from the Annual Huntington's Disease Therapeutics Conference.
A new clinical trial just announced for 2015 aims to test a “huntingtin lowering” therapy, called an antisense oligonucleotide (ASO), that attacks mutant huntingtin directly. We’re extremely excited—it’s the first-ever human HD trial to fight HD at the root of the problem, and has shown great promise in animal models. What’s the scoop?
Pharmaceutical giant Roche recently described a new drug delivery technology they call the 'brain shuttle'. Why has Huntington's disease been mentioned in so many press releases about this technology, and how much can we hope to gain from this new advance?
Our final report from the last morning of the World Congress on Huntington's Disease in Rio de Janeiro, Brazil.
Gene silencing drugs, which tell cells to stop making the harmful huntingtin protein, are among the top approaches being worked on to fight Huntington's disease. A human trial in motor neuron disease using 'ASO' gene silencing drugs has just shown the drugs and delivery method to be safe, boosting plans to get clinical trials of these drugs up and running in HD.
Our second daily report from the annual Huntington's Disease Therapeutics Conference in Venice, Italy. You can tweet @HDBuzzFeed, comment on Facebook or use HDBuzz.net to send us questions, comments and queries.