Gene therapy trial aims to restore lost skills in rett syndrome
Disease control
Recruiting now
This study tests a new gene therapy called NGN-401 in females with Rett syndrome, a rare genetic disorder that affects development and movement. The goal is to see if the treatment can help participants gain new skills or improve their condition. About 33 girls and women aged 3 a…
Phase: PHASE3 • Sponsor: Neurogene Inc. • Aim: Disease control
Last updated May 11, 2026 20:47 UTC