$8 million awarded to study brain cell death in fatal pediatric diseases, including Sanfilippo

October 5, 2023

Washington University School of Medicine (St. Louis, MO) has received nearly $8 million from the National Institute of Neurological Disorders and Stroke of the National Institutes of Health (NIH) to help determine the root causes of brain cell death in fatal pediatric neurodegenerative diseases.

A wide-ranging multidisciplinary team will focus on Sanfilippo syndrome and Late Infantile Batten Disease, two fatal lysosomal diseases in which children experience severe neurodegeneration. The innovative project will explore, in detail, the ways diseased cells communicate with one another and those relationships to cell death, potentially uncovering new treatment strategies.

“The funded research aims to lay a foundation for the development of therapies that could potentially halt brain cell death,” stated the university in a press release.

Cure Sanfilippo Foundation was glad to be able to provide a letter of support for this important project which was submitted as part of the team’s NIH application process.

“Huge congratulations to the Washington University team and Dr. Patricia Dickson! We look forward to collaborations with the team in support of this important research,” said Foundation President & Co-Founder Glenn O’Neill.

The Washington University team includes Dr. Patricia Dickson, who recently presented at the ADVANCE 2023, Sanfilippo Community Conference. Her presentation, “Membrane-tethered NAGLU to explore origins of CSF heparan sulfate,” is available for on-demand watching on the Foundation’s website.

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