Summary
- Participate in HD research: Online surveys, observational studies, & more
- Overcoming the bumps on the way to HD therapies
- FDA grants orphan drug designation to SAGE-718
Participate in HD research: Online surveys, observational studies, & more
There are dozens of opportunities to participate in HD research that you can explore from home or with the help of your medical provider. HD Trialfinder is a great place to get started seeking HD research opportunities; create a profile and match to studies to see what options may be available to you. HDSA’s online surveys page is also a great place to contribute your voice to studies from the comfort of your own home.
Additionally, Design Science is seeking opinions from people with HD and their partners about a new medical device used for swallowing medications. The current study is taking place in Philadelphia and Chicago, and it involves a 45-minute paid, in-person interview. There is no medication or procedure; it simply involves looking at the device and answering some questions. Compensation is $350 for a person with HD, and an additional $125 for their accompanying partner. This is open to people diagnosed with HD at any stage. You can sign up for this study at www.dscience.com/signup, filling out your info, and writing “Huntington’s disease” in the box that asks what you want to learn more about. You can also email ihaveavoice@dscience.com.
Overcoming the bumps on the way to HD therapies
At last week’s HDSA Education Day in San Diego, UCSD Center of Excellence Director Dr. Jody Corey-Bloom provided an update on HD care and research that explained HD genetics, treatments, and the current drug pipeline in simple terms. Local advocate and HD blogger Ken Serbin, aka “Gene Veritas,” captured the talk on video, and it’s available to view here.
FDA grants orphan drug designation to SAGE-718
Sage Therapeutics announced late last week that the FDA has granted Orphan Drug Designation (ODD) to SAGE-718, a drug that is being tested in clinical trials to treat the cognitive (thinking) symptoms of HD. ODD is granted to support the development of drugs for safe and effective treatment of orphan diseases. It provides financial incentives that encourage companies to focus on rare diseases, like HD. Sage’s clinical studies are ongoing through the Perspective Program. Although ODD doesn’t typically affect the pace of clinical research, it represents a milestone in drug development. To read the Sage community letter, click here.
