This study tests a gene therapy called GS-100 for children aged 2 to 18 with NGLY1 deficiency, a rare genetic disease. The treatment is given once into the fluid around the brain. The goal is to see if it safely improves motor skills and development. Ten participants will be foll…
Phase: PHASE3 • Sponsor: Grace Science, LLC • Aim: Disease control
Last updated May 08, 2026 19:30 UTC