When you have a rare disease, you face two battles – one being the illness itself, and the other, living in a world where so few people understand what you’re up against. Huntington’s disease is a rare disease and the HD community is no stranger to these battles, but we also know firsthand that while we may be rare, we are mighty and we are getting stronger together each day. Monday, February 28th is Rare Disease Day 2022, and HDSA is joining forces with individuals from over 7,000 rare diseases affecting 300 million people worldwide to raise awareness about rare disorders like HD. Check out https://www.rarediseaseday.org/ to learn more about the rare disease community and the events happening worldwide. Visit www.hdsa.org/rarediseaseday to join us in turning up the global volume for Huntington’s disease awareness and to support HD research and world-class HD resources for families across the country.
HDBuzz covers a new tool to track the progression of HD
An international collaboration of scientists has set out to find new ways to track HD. HD is caused by an error in the huntingtin gene that causes mutant huntingtin protein, mHTT, to be produced by the body. Over time, mHTT accumulates in the body and forms toxic clumps. Using a chemical tool to flag these clumps, called a PET ligand, scientists were able to visualize how mHTT levels change in HD animal models. These flags can be seen with non-invasive imaging technologies, which means they could potentially be used to track the effects of huntingtin–lowering therapies. To learn more about this exciting new tool, read more from HDBuzz here.
Sage Therapeutics opens enrollment for DIMENSION study of SAGE-718
Sage Therapeutics announced this week that they will open enrollment for the DIMENSION study, a Phase 2 clinical research study for Sage’s investigational drug, SAGE-718. This first-in-class drug targets cognitive decline in HD and related neurodegenerative diseases. SAGE-718 received FDA fast-track designation last September, a process designed to facilitate the development and review of new treatments for serious conditions with unmet medical need like HD. Study sites around the USA will begin recruiting participants in the near future; keep your eye on HDTrialfinder.org for updates and to learn more.