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 17, 2026 06:20 UTC
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Brain imaging breakthrough could save babies from rare disease
Diagnosis OngoingThis study looks at whether a special type of MRI called diffusion tensor imaging (DTI) can help doctors identify newborns with Krabbe disease who need urgent treatment. Researchers will scan the brains of 100 infants who tested positive for the disease and track their motor deve…
Matched conditions: KRABBE DISEASE
Sponsor: University of Pittsburgh • Aim: Diagnosis
Last updated May 04, 2026 16:19 UTC