One-Time gene injection aims to slow rare fatal brain disease in kids
Disease control
Ongoing
This early-stage study tests a one-time gene therapy injection into the spinal fluid of children aged 1-18 with CLN7 Batten disease, a rare and fatal genetic brain disorder. The main goal is to check safety, but researchers will also measure changes in movement, thinking, and vis…
Phase: PHASE1 • Sponsor: Benjamin Greenberg • Aim: Disease control
Last updated Jun 27, 2026 08:07 UTC