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Exciting News in Huntington’s Disease Research

By May 14, 2019Commentary, In the News

Contributed by Joan Steffan, PhD & Leslie Thompson, PhD

Results from a recent study published by Tabrizi and colleagues and Ionis/Roche Pharmaceuticals in The New England Journal of Medicine are very exciting for the Huntington’s disease (HD) patient, family, and scientific communities. The researchers showed for the first time that treatment with a huntingtin lowering drug called an antisense oligonucleotide, or ASO, is safe in HD patients.  Huntingtin is the protein linked to the genetic mutation that causes HD. With these results, researchers are planning a large clinical trial to test whether ASO can reduce symptoms in HD patients. The HD community is hopeful this therapy may be the first effective treatment to slow HD progression.

In other exciting news, UCI MIND HD scientist Dr. Leslie Thompson and her team were awarded $6 million by the California Institute for Regenerative Medicine to perform continued testing of promising stem cell treatments for HD (UCI News). To learn more about HD research and care at UCI, visit the website of our partner organization HD-CARE at