Page 1 Page 2 Page 3 Page 4 Page 5 Page 6 Page 7 Page 8 Page 9 Page 10 Page 11 Page 12 Page 13 Page 14 Page 15 Page 16 Page 17 Page 18 Page 19 Page 20Let’s reflect on all the exciting progress that has been generated in the HD research community over the past year. In 2016, HD clinical trials from Teva, Pfizer and Vaccinex all completed their recruitment. The Enroll-HD registry and observational platform enrolled its 10,000th participant. A novel new drug to combat the motor symptoms associated with HD was submitted to the FDA for approval. Ionis Pharmaceuticals announced that they have started administering the highest and final dose of their antisense oligonucleotide (ASO) to early stage HD patients in the first of its kind study to investigate a huntingtin lowering drug. Finally, a new study was initiated by Azevan here in the US to see if their drug could improve irritability and aggression in HD patients. Research and care programs at the Huntington’s Disease Society of America demonstrated great progress in 2016 as well. HDSA expanded the network of Centers of Excellence to 39 across 30 states. We continued to expand our patient-focused research commitments and launched a call center for HD TrialFinder to provide families more support as they consider which clinical trials to take part in. In just four years, HDSA has now committed more than $3 million to the HD Human Biology Project. The Human Biology Project is a critical piece of HDSA’s mission to support impactful HD research that will help guide us closer to effective therapies. The research we support is all patient-centric and done in collaboration with HD clinics from around the globe with the goal of understanding HD in the only place it naturally occurs, in humans. In this report you can find summaries of the nine new research projects HDSA is supporting. A new research grant program — the Berman- Topper Family HD Career Development Fellowship — was introduced in late 2015 to provide young scientists with the time and resources to allow them to transition into an independent HD investigator. These achievements all have one thing in common. They could not have been achieved without the participation of HD families. We are indebted to the brave HD research heroes around the world who volunteer selflessly so that we will not have to wait another generation for effective HD treatments. Today, there are approximately 10 companies actively pursuing different approaches to reduce huntingtin, the root cause of HD. While the tech- nologies hold great promise, the only way to figure out if they will work for HD is to test them in people. 2017 and beyond will provide many additional opportunities to get involved in HD clinical trials. We have no doubt that the HD community will continue to rise to these challenges. I am confident you will agree with me that in 2016 HDSA and the research community have made tremendous progress towards meaningful clinical trials that will hopefully modify the course of HD and bring relief and hope to you — our HD families. George Yohrling, Ph.D. HDSA Senior Director, Mission & Scientific Affairs In HD Science, the Momentum Is Building RESEARCH REPORT 11