Experimental gene therapy aims to improve motor skills in krabbe children
NCT ID NCT05739643
First seen Nov 20, 2025 · Last updated May 16, 2026 · Updated 20 times
Summary
This study tests a one-time gene therapy (FBX-101) given through an IV to children with Krabbe disease who have already received a stem cell transplant. The goal is to see if it is safe and can improve motor function like walking and coordination. Nine participants will receive the therapy, and their progress will be compared to similar patients who only had the transplant.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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