Doctors test One-Time gene fix for devastating childhood brain disease
NCT ID NCT03727555
Summary
This trial is testing a new gene therapy for X-linked adrenoleukodystrophy (X-ALD), a severe genetic brain disease that often affects young boys. Researchers are giving patients a one-time treatment that delivers a corrected gene directly into the body, aiming to fix the underlying genetic cause. The main goals are to see if this approach is safe and if it can slow or stop the progression of the disease.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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