Experimental gene therapy aims to halt rare childhood disease
NCT ID NCT06199531
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
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, with safety and effectiveness tracked over a year.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Oakland Children's Hospital (UCSF Benioff)
Oakland, California, 94609, United States
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Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, 77030, United States
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