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Batten Disease Press Releases

One-and-done gene therapy

Batten disease may benefit from gene therapy
Wednesday, Nov 11, 2015
In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder.

Four New Members Appointed to National Neurological Disorders and Stroke Advisory Council
Thursday, Feb 4, 2010
Four New Members Appointed to National Neurological Disorders and Stroke Advisory Council

Study Finds Biochemical Defect in Juvenile Batten Disease
Wednesday, Jan 25, 2006
For the first time, scientists studying a fatal childhood neurodegenerative disorder, juvenile Batten disease, have identified a defect in transport of the amino acid arginine in cells from affected children. The finding helps researchers understand how the disease develops and may lead to new ways of treating it.

Study Finds Autoimmune Link In Juvenile Batten Disease
Wednesday, May 22, 2002
For years, researchers have tried to determine how the defective gene in juvenile Batten disease leads to the seizures, mental impairment, and other symptoms of this devastating childhood disorder. A new study shows that mice lacking the gene that is altered, or mutated, in this disorder have an immune reaction that disables an important enzyme in the brain. The study also found signs of this reaction in children with Batten disease. The finding provides a new clue about how Batten disease may damage the nervous system and could lead to treatments for the disorder.
Fact Sheet

Gene for Last Major Form of Batten Disease Discovered
Friday, Sep 19, 1997
Just two years ago, the origins of the fatal childhood neurological disorders called Batten disease were shrouded in mystery, and there were few prospects for effective treatment. Now, for the first time, researchers can describe the genetic underpinnings of all major childhood forms of the disease.