Batten Disease

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.