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GoodRx Guide

Batten Disease

Comprehensive information for you or a loved one — including treatment options and discounts on popular medications.

Batten Disease refers to a group of rare, inherited disorders of the nervous system. It is caused by a mutation in the gene and affects a cell's ability to recycle certain molecules. This causes a buildup of fats in the cells of the brain, nervous system, and retina.

Novel drug Brineura is the only FDA approved treatment that treats a specific form of Batten disease. Other symptoms such as seizures can be improved with medications.

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Brineura

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