Gene therapy injection aims to slow rare childhood brain disease
Disease control
Ongoing
This early-stage study tests a single injection of gene therapy into the spinal fluid of children with CLN7 Batten disease, a rare and severe genetic brain disorder. The main goal is to check if the treatment is safe, but researchers will also look for any signs it helps with mov…
Phase: PHASE1 • Sponsor: Benjamin Greenberg • Aim: Disease control
Last updated May 01, 2026 16:00 UTC