Diseases Studied

Batten Disease is a group of rare, inherited nervous system disorders. It is caused by genetic changes that disrupt the body’s ability to remove cellular waste, leading to a buildup of harmful materials in brain and nerve cells. Over time, this causes progressive neurological decline, including vision loss, seizures, cognitive impairment, motor difficulties, and often shortened lifespan.

Batten Disease is also called neuronal ceroid lipofuscinoses (NCLs). There are 13 recognized NCLs, which vary by age of onset, symptoms, and rate of progression depending on the specific gene involved.