This study tests whether injecting a replacement enzyme directly into the eye can slow or stop vision loss in children with CLN2 Batten disease, a rare genetic disorder that causes blindness and brain damage. Five children aged 2 to 6 who already receive enzyme therapy into the b…
Phase: PHASE1, PHASE2 • Sponsor: David L Rogers, MD • Aim: Disease control
Last updated May 06, 2026 16:14 UTC