Experimental drug tested for rare childhood brain disease in adults

This study tested the safety and effects of an oral drug called miglustat in six adults (age 17+) with CLN3 Batten disease, a rare and serious genetic disorder that affects the brain. Participants took the drug daily for two years while researchers monitored for side effects, how the drug moved through their bodies, and whether it helped control symptoms like seizures or movement problems. The goal was to see if miglustat could help manage this difficult disease.