what is cln1 (batten disease)
CLN1 Batten Disease is a rare, inherited neurodegenerative disorder that primarily affects young children. Caused by mutations in the CLN1 gene, this condition prevents the body from producing a critical enzyme that helps clear waste from cells—especially in the brain and nervous system. As a result, children experience a heartbreaking progression of symptoms, including vision loss, seizures, motor and cognitive decline, and eventually, loss of basic functions. There is currently no cure. But there is hope. Through advocacy, research, and the courage of families like Jack’s, we are fighting to change what the future looks like for children facing CLN1.
life without a cure
There are currently no FDA-approved treatments for CLN1 Batten Disease. Families are left with supportive care only—managing symptoms as they progress while watching their child lose skills they once had. It’s a devastating road marked by fear, exhaustion, and heartbreak. Every day becomes a delicate balance of hope and grief, of celebrating small victories while facing the unimaginable. For families like ours, time is everything. That’s why we are pushing for access to experimental treatments, advocating for faster approvals, and working tirelessly to give children like Jack a fighting chance.