Gene therapy trial offers hope for kids with rare brain disease
NCT ID NCT07236606
First seen Nov 19, 2025 · Last updated May 14, 2026 · Updated 20 times
Summary
This study tests a gene therapy called RGX-121 in children with Hunter syndrome, a rare genetic disorder that affects the brain and body. The therapy delivers a working copy of the missing gene to help improve brain development and reduce harmful substance buildup. The trial is for boys aged 4 months to under 5 years with the severe form of the disease. Researchers are checking if the treatment is safe and effective over several years.
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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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Locations
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St. Peter's University Hospital
New Brunswick, New Jersey, 08901, United States
Conditions
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