NGLY1 DEFICIENCY

Clinical trials for NGLY1 DEFICIENCY explained in plain language.

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Gene therapy offers hope for kids with rare NGLY1 disorder

Disease control Ongoing

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…

Matched conditions: NGLY1 DEFICIENCY

Phase: PHASE3 • Sponsor: Grace Science, LLC • Aim: Disease control

Last updated May 08, 2026 21:15 UTC

NGLY1 DEFICIENCY

Get alerted when new NGLY1 DEFICIENCY trials appear

Gene therapy offers hope for kids with rare NGLY1 disorder