Brain-Delivered gene therapy offers hope for devastating childhood disorder
NCT ID NCT07270549
Summary
This is the first human trial of a gene therapy for children with CTNNB1 syndrome, a severe genetic disorder that affects brain development. Researchers will test if a single dose of a therapy called Urbagen, delivered directly to the brain, is safe and can improve motor function, thinking skills, and quality of life. The study will enroll 12 children aged 2-12 years and follow them for up to five years.
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Contact
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Locations
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University Medical Centre Ljubljana
RECRUITINGLjubljana, 1000, Slovenia
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Conditions
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