Gene therapy trial halted: hope for kids with rare brain disease
NCT ID NCT07236606
First seen Nov 19, 2025 · Last updated May 22, 2026 · Updated 21 times
Summary
This study tests a gene therapy called RGX-121 for children with Hunter syndrome, a rare genetic disease that affects the brain and body. The therapy aims to deliver a working gene to the brain to help improve development. The trial is currently suspended and enrolled only 2 children to check safety and effectiveness.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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St. Peter's University Hospital
New Brunswick, New Jersey, 08901, United States
Conditions
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