This study tests a one-time gene therapy called GS-100 in 10 children aged 2 to 18 with NGLY1 deficiency, a rare genetic disorder. The therapy is given directly into the brain fluid to deliver a working copy of the missing gene. The goal is to improve motor skills and development…
Phase: PHASE3 • Sponsor: Grace Science, LLC • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC