One-Time brain injection aims to halt devastating genetic disorder in girls
Disease control
Recruiting now
This study is testing a one-time gene therapy called NGN-401 for girls with Rett syndrome, a severe genetic disorder that impairs brain development. The therapy delivers a working copy of a missing gene directly to the brain. Researchers will follow participants for 3 years to se…
Phase: PHASE3 • Sponsor: Neurogene Inc. • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC