Gene therapy hope for kids with rare citrate disorder
Disease control
Recruiting now
This early-stage trial tests a single dose of TSHA-105, a gene therapy injected into the spinal fluid, in 8 people aged 2 to 20 with SLC13A5 citrate transporter disorder. The goal is to see if it is safe and whether it can improve motor and thinking skills. Because the trial is v…
Phase: PHASE1, PHASE2 • Sponsor: TESS Research Foundation • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC