One-Shot gene therapy trial offers hope for devastating childhood disorder
Disease control
Not yet recruiting
This study is testing a single injection of an experimental gene therapy called TSHA-102 in three young girls with Rett syndrome. The goal is to see if the treatment is safe and if it can help control the disease by targeting its genetic cause. Participants will be followed close…
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated Mar 23, 2026 15:16 UTC