One-Time gene therapy trial offers hope for fatal childhood brain disease
NCT ID NCT03952637
Summary
This study is testing a one-time gene therapy for children with GM1 gangliosidosis, a rare and fatal genetic disorder that destroys nerve cells. The therapy aims to deliver a working copy of a missing gene to help the body produce a vital enzyme. Researchers will assess the safety of the treatment and whether it can slow or improve symptoms in infants and young children.
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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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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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