CLN7

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Not yet finished but already full! 1

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…

Matched conditions: CLN7

Phase: PHASE1 • Sponsor: Benjamin Greenberg • Aim: Disease control

Last updated Jun 27, 2026 08:07 UTC

CLN7

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One-Time gene injection aims to slow rare fatal brain disease in kids