Gene therapy offers hope for kids with rare NGLY1 disorder
NCT ID NCT06199531
First seen May 05, 2026 · Last updated May 23, 2026 · Updated 5 times
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 condition. The therapy is given directly into the brain fluid to deliver a working copy of the missing gene. Researchers will check if it safely improves motor skills and development over a year.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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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
Conditions
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