This week, Dr. Fox returns from maternity leave and resumes the HDSA research blog!

The 34th Annual HDSA Convention in Boston was a great success, with the largest attendance on record (1,225 guests). Huntington’s disease research had a strong presence, with talks from HDSA fellows, sessions on gene therapy, research Q&As, and representatives from biotech and pharma. Here are some of the highlights:

Drs. Ed Wild and Jeff Carroll, HD researchers and editors of HDBuzz talked all things science with the National Youth Alliance as well as during their ever-popular “Ask a Scientist Anything” session. During Saturday’s research forum they also provided a fabulous recap of the Huntington’s disease research landscape, recorded here, in which they borrowed themes from the Avengers: Endgame movie to describe the important themes and pursuits in HD research right now.

Dr. Robert Pacifici, Chief Scientific Officer at CHDI, discussed how families can critically contribute to HD research. You can watch the recorded session at this link.

Saturday’s research forum featured Dr. Craig Mello, who won the Nobel Prize for his discovery of RNAi, and Dr. Anastasia Khvorova, who is using RNAi approaches to develop therapies for Huntington’s disease. Their dynamic talk was also live streamed and can be viewed here.

Additional sessions showcased biotech companies from the Boston area working on Huntington’s disease (there are many!), and company representatives spoke with families in the exhibit hall. Roche/Genentech and Wave discussed their ongoing clinical trials, and representatives from Vybion and PTC Therapeutics discussed novel huntingtin-lowering therapies in the works.


Several sessions featured HDSA Human Biology Fellows. Dr. Wasim Malik talked about his work on tracking eye movements to detect early movement changes in HD, while Dr. Sophie Andrews shared her findings that moderate exercise can be helpful for boosting memory in people with the HD gene. Dr. Edith Pfister reviewed current approaches to gene therapy, and 2018 Berman-Topper Fellow Dr. Rachel Harding spoke about the hunt for huntingtin’s binding partners – which other molecules it hangs out with in our cells. Donald King research fellows Ethan Smith, Jacob Friedman, Scott Song, and Wes Solem, as well as high school student and Intel Finalist Ritika Jeloka presented posters on their HDSA-funded summer projects in HD laboratories.

Changing laws and rising interest in alternative treatments for HD symptoms led to this year’s session on medical cannabis for Huntington’s disease, in which three researchers provided an overview of the pharmacology of cannabis and discussed the limited human research from a pro/con perspective.

Finally, during Convention, UniQURE issued an exciting letter to the community about their upcoming clinical trial to test a gene therapy for HD.

To all those eager for HDSA research updates: welcome back to the blog, and happy summer!