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Batten disease, also known as neuronal ceroid lipofuscinoses, is a group of progressive conditions impacting the nervous system. Batten disease is caused by mutations in several different genes that are important for the proper functioning of lysosomes. Lysosomes are the compartments in cells that break down molecules. When lysosomes don't work properly it leads to a buildup of toxic materials in the cells of the body. This causes the symptoms of Batten disease, which may include vision loss, seizures, abnormal movements, cognitive problems, and developmental regression. Most forms of Batten disease onset in children and result in significantly reduced life expectancy. There are also adult-onset forms which tend to have milder symptoms. There is no cure for Batten disease and only one subtype has an approved treatment.
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We are jumpstarting our Batten disease program to accelerate research on the condition and welcome all parents and caregivers interested in participating and spreading the word to the Batten disease community.