Gene therapy trial aims to help girls with rett syndrome gain new skills
Disease control
Ongoing
This study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whe…
Phase: PHASE3 • Sponsor: Neurogene Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC