CLN3 research hub

Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease

This clinical trial is testing a liquid medicine called Batten-1 (miglustat) for children and teenagers with CLN3 Batten disease. Researchers want to see if taking this medicine every day can help slow down or stop vision loss, while checking for any side effects and monitoring overall wellbeing.