New hope for batten disease: experimental drug tested in adults
Disease control
Completed
This study tested the safety and effectiveness of the drug miglustat in 6 adults aged 17 and older with CLN3 Batten disease, a rare genetic disorder that damages the nervous system. Participants took miglustat for up to 104 weeks to see how the body processes the drug and whether…
Phase: PHASE1, PHASE2 • Sponsor: Beyond Batten Disease Foundation • Aim: Disease control
Last updated May 26, 2026 12:07 UTC