Experimental drug tested for rare brain disease in adults

This study tested whether an oral drug called miglustat is safe and might help control symptoms in adults with CLN3 Batten disease, a rare genetic disorder that affects the brain. Six participants aged 17 and older took the drug daily for two years while researchers monitored side effects, drug levels in the blood, and changes in symptoms like movement problems and seizures. The goal was to find the highest dose people could tolerate and see if it showed any benefit for this currently incurable condition.