Gene therapy offers hope for kids with rare NGLY1 disorder
NCT ID NCT06199531
First seen May 05, 2026 · Last updated May 08, 2026 · Updated 3 times
Summary
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 followed for one 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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