New gene therapy aims to halt devastating nerve disease in children
NCT ID NCT05739643
Summary
This study is testing a one-time gene therapy called FBX-101 for children with Krabbe disease, a rare and severe genetic disorder that damages the nervous system. The therapy is given as an infusion to children who have already received a stem cell transplant, which is the current standard treatment. Researchers hope this added gene therapy will help improve children's movement and motor skills beyond what the transplant alone can achieve.
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Contacts and locations
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Locations
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Children's Hospital of Orange County (CHOC)
Orange, California, 92868, United States
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Duke University Medical Center
Durham, North Carolina, 27705, United States
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University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109, United States
Conditions
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