National Gaucher Foundation Commends NIH for Continued Research on Gaucher Disease

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National Gaucher Foundation today commended National Institutes of Health scientist Dr. Ellen Sidransky for her tireless work on Gaucher disease. Her research, which was published on July 12, identified a molecule that could lead to new treatment methods for the disease.


NIH-backed research has already had a tremendous impact on the lives of thousands who suffer from Gaucher disease, like me.

National Gaucher Foundation today thanked the National Institutes of Health for the agency’s continued research on Gaucher disease. The statement follows an announcement that researchers have made advancements in possible treatments for the rare lysosomal storage disease.

The Journal of Neuroscience published findings by NIH’s Ellen Sidransky, M.D. and her partners at National Institute of Neurological Disorders and Stroke and the National Center for Advancing Translational Sciences on July 12. Their research identified a molecule that could be used to treat Gaucher disease by breaking down the build-up of lipids in the brain.

Dr. Sidransky has been researching Gaucher disease, which is estimated to be present in one of every 75,000 live births, for 28 years. In addition to her work at NIH, she also sits on National Gaucher Foundation’s Medical Advisory Board.

“NIH-backed research has already had a tremendous impact on the lives of thousands who suffer from Gaucher disease, like me,” said National Gaucher Foundation President Brian Berman. “Though there is a long way to go before we know if this research will lead to a new treatment, we are thrilled that Dr. Sidransky and her colleagues have been so focused on enhancing the lives of people living with Gaucher disease.”

While at NIH, the late Dr. Roscoe Brady developed the first treatment for Gaucher disease in the 1980s. Dr. Sidransky’s research builds on that proud tradition, and could one day help treat patients with Parkinson’s disease as well.

About National Gaucher Foundation

The National Gaucher Foundation was established in 1984 with three goals: to find the cause of Gaucher disease, to develop a cure and to promote research and awareness of the disease. Following the determination of the cause and the creation of a treatment, the Foundation expanded its programs to include education and financial assistance for those living with Gaucher disease. Today, the National Gaucher Foundation is the only independent, not-for-profit educational organization in America that caters to individuals and families with the disease. To learn more about Gaucher disease or the work of the National Gaucher Foundation, visit

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