One-Shot gene therapy aims to halt devastating brain disease in toddlers
NCT ID NCT07236606
Summary
This study is testing a one-time gene therapy called RGX-121 for young boys with a severe form of Hunter syndrome that affects the brain. The goal is to see if delivering a working gene directly to the brain can slow or stop the loss of thinking and developmental skills. The study will measure changes in development, brain scans, and safety for up to five years.
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Contacts and locations
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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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