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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.

2024 Boston Marathon presented by Bank of America

This year's event took place on Monday, April 15, 2024, and the UMass ALS Cellucci Fund had 10 runners! 

Meet the 2024 Boston Marathon team

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UMass Chan clinical trial shows antisense oligonucleotide safely suppresses mutant ALS gene in pilot human study

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.

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