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 brain infusions of the en…
Phase: PHASE1, PHASE2 • Sponsor: David L Rogers, MD • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC