Experimental drug miglustat tested for rare batten disease
Disease control
Completed
This study tested the drug miglustat in 6 adults aged 17 and older with CLN3 Batten disease, a rare genetic disorder that causes nerve damage. The goal was to see if the drug is safe and to find the best dose over 2 years. Researchers tracked side effects and how the drug moved t…
Phase: PHASE1, PHASE2 • Sponsor: Beyond Batten Disease Foundation • Aim: Disease control
Last updated Jun 26, 2026 14:56 UTC