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How Iowa and Rutgers are taking down a rare and devastating disorder: BTN LiveBIG

Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) belongs to a family of disorders more commonly known as Batten Disease, although it would be a stretch to say that Batten Disease is at all commonly known. It only occurs in about two to four births out of every 100,000 in the United States. Its effects, though, are devastating. In people with CLN2, an aggressive form of Batten Disease that first presents around 2-4 years old, a defective gene inhibits production of a crucial enzyme which allows brain cells to rid themselves of waste. The resulting buildup leads to widespread cell death. Symptoms