Huntington’s disease was featured this week at the American Academy of Neurology (AAN) Conference, where 12,000 medical professionals gathered for a yearly forum on neurology research. The mainstage presentation, in front of a huge audience of doctors and scientists, featured the most recent positive results from the Phase 1b/2a trial of the huntingtin-lowering drug from Ionis Pharmaceuticals. In addition to the promising safety and huntingtin-lowering results that were shared in early March, Ionis showed some early, tentative evidence that huntingtin lowering is associated with improvements in symptoms. However, these findings are only exploratory, and will need to be confirmed in the larger phase 3 trial that is in the planning stages.

Because the results were in the spotlight during a highly-attended session at the AAN Conference, it’s likely to make a big splash in the news. On Friday Roche (the company taking over the next phase of the trial) released a statement expressing their gratitude to the HD community, and summarizing the results and what’s happening next. Essentially, they are working hard at the massive task of planning Phase 3, and will continue to present the data within the scientific and medical community. We’re excited that the drug continues to show great promise, and the wide exposure at international meetings will positively impact families by raising awareness of HD among medical professionals worldwide.


Four young scientists selected for HDSA’s Donald A. King Summer Research Fellowships

We’re pleased to announce the four winners of this year’s Donald A. King Summer Research Fellowships, who were selected from a fantastic pool of applicants after rigorous review by HDSA’s scientific advisory board. To learn more about the program and about the work these undergraduate students will conduct in laboratories over the summer of 2018, check out our press release.