Tracking the future: Long-Term watch on pioneering gene therapy for devastating childhood disease
NCT ID NCT06308718
Summary
This study is monitoring children with Krabbe disease who previously received an experimental gene therapy called FBX-101 in earlier trials. The goal is to track their long-term safety and see how their motor skills, thinking, and overall health change over several years. It is an observational study, meaning the children are not receiving new treatment, but their health is being carefully followed.
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Contacts and locations
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Locations
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University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109, United States
Conditions
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