Supporting the boldest research ideas in the interdisciplinary labs under the direction of Robert H. Brown Jr., DPhil, MD, one of the world's foremost authorities on ALS.
This year's event will take place on April 21, 2025. Five runners are fundraising on behalf of the UMass ALS Cellucci Fund!
Using a short, synthetic chain of chemically modified nucleotides engineered in the RNA Therapeutics Institute at UMass Chan Medical School, Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of the ALS (amyotrophic lateral sclerosis) gene known as C9ORF72 in a single-patient pilot study. C9ORF72 is the most common cause of familial ALS and familial frontotemporal dementia (FTD). The results, published in Nature Medicine, have the potential to catalyze research into treatments for ALS, FTD and other neurodegenerative diseases.
