Gene therapy hope for toddlers with rare brain disorder
Disease control
Not yet recruiting
This study tests a single dose of a gene therapy called TSHA-102 in 3 girls aged 2 to 4 with Rett syndrome, a severe genetic disorder. The therapy is given as a spinal injection and aims to improve symptoms by delivering a working copy of the faulty gene. The main goal is to chec…
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated May 12, 2026 13:40 UTC