In case you have missed it: The results of the Ionis phase I huntingtin-lowering trial were announced on 11 December 2017 and appear very promising (BBC, The Guardian, CNN). The antisense oligonucleotide-based drug, IONIS-HTTRx, not only appeared to be safe and well-tolerated, but according to the company’s press-release, also dose-dependently decreased mutant huntingtin levels which “substantially exceeded expectations”. Grateful for having been able to make a small contribution to this major breakthrough as part of Professor Sarah Tabrizi’s clinical research team. We have now started the open lable extension study and I am very hopeful that we have taken a large step forward in finding a treatment for this devastating disease, although we are not there yet!
Curious Brain 