Gene therapy trial offers hope for girls with rare brain disorder
Disease control
Ongoing
This study tests a new gene therapy called TSHA-102 for girls aged 5 to 8 with Rett syndrome, a serious genetic disorder that affects brain development. The therapy is given as a single injection into the spinal fluid to try to improve symptoms. The main goal is to check safety, …
Phase: PHASE1, PHASE2 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated May 17, 2026 06:48 UTC