New gene therapy hopes to help girls with rare brain disorder
Disease control
Recruiting now
This study tests a one-time gene therapy called TSHA-102 for females with Rett syndrome, a rare genetic disorder that affects brain development. The therapy is given as a single injection into the spinal fluid. The goal is to see if it is safe and can help improve skills like wal…
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated May 17, 2026 11:38 UTC