KRABBE DISEASE
Clinical trials for KRABBE DISEASE explained in plain language.
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Experimental gene therapy aims to improve motor skills in krabbe children
Disease control OngoingThis 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 t…
Matched conditions: KRABBE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated May 18, 2026 12:13 UTC
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Brain imaging breakthrough could save babies from rare disease
Diagnosis OngoingThis study uses a special MRI technique called diffusion tensor imaging (DTI) to look at brain development in newborns with Krabbe disease, a rare and serious genetic disorder. The goal is to find which babies need immediate treatment to prevent severe disability. Researchers wil…
Matched conditions: KRABBE DISEASE
Sponsor: University of Pittsburgh • Aim: Diagnosis
Last updated May 22, 2026 13:53 UTC