A study recently published in Nature detailed scientists’ understanding of how branaplam, a drug originally developed for treatment of spinal muscular atrophy (SMA), may be used to treat HD. HDBuzz gave us the highlights: A group of researchers at Novartis and The Children’s Hospital of Philadelphia tested the drug’s effects in cells in a dish as well as in a mouse model of HD. They found that branaplam lowered levels of huntingtin protein by interfering with the message that codes for the protein, and showed early evidence that it could improve the performance of HD mice on a movement test. Finally, the researchers looked at samples from individuals with SMA who had participated in a clinical trial for branaplam, and found that huntingtin levels went down and stayed down throughout the trial. All of this lends support to the ongoing safety trial of branaplam being conducted in people with HD. Read the full report from HDBuzz here.
Brain Awareness Week 2022
Brain Awareness Week (March 14th-20th, 2022) is a global campaign to foster public enthusiasm and support for brain science, coordinated by the Dana Foundation. Every March, partners host imaginative activities in their communities that share the wonders of the brain and the impact brain science has on our everyday lives. Check out www.brainawarenessweek.org to learn more about virtual events all over the world and find resources for all ages to help kids and adults learn more about the brain and neuroscience research.
One driving force behind research on the human brain is the availability of human brain tissue. A brain donation is an invaluable gift anyone can make to further how we understand the brain and different diseases that affect it, like HD. Learn more about brain donation on HDSA’s website, here.
HDSA staff talks research at HDYO Virtual Congress
HDYO, an international nonprofit dedicated to supporting, educating, and empowering young people affected by HD, recently held its International Young Adults Congress that brought together young people around the world to talk about all things HD. The virtual gathering featured talks from numerous leaders in HD Research. Recordings of these sessions can be found on HDYO’s YouTube channel, including a panel discussion between HDSA’s Arik Johnson, PsyD, Leora Fox, PhD, and HDSA Berman-Topper Fellow Lauren Byrne, PhD, covering hot topics in HD research and exciting therapeutic approaches on the horizon. Watch the full conversation here.