Gene Therapy Brings Hope to Children with Batten Disease

Batten disease is a tragically devastating disease so rare that only about 200 children around the world have it at any one time. One reason for its rareness is that it most often kills the stricken child before his or her 12th birthday. Also known as LINCL, or Late Infantile Neuronal Ceroid Lipofuscinosis, the genetic disease hinders the child’s brain cells’ ability to expel cellular waste material. The ever-expanding amount of cellular waste clogs the cell, becoming toxic, and eventually causing enough damage to kill the cells in the brain. Read more