Experimental drug tested for rare childhood brain disease in adults
Disease control
Completed
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…
Phase: PHASE1, PHASE2 • Sponsor: Beyond Batten Disease Foundation • Aim: Disease control
Last updated Mar 16, 2026 15:27 UTC