KRABBE DISEASE
Clinical trials for KRABBE DISEASE explained in plain language.
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New gene therapy aims to halt devastating nerve disease in children
Disease control OngoingThis 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 curren…
Matched conditions: KRABBE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated Mar 30, 2026 14:34 UTC
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Race against time: brain scans could save babies from rare deadly disease
Diagnosis OngoingThis study aims to use a special brain scan called diffusion tensor imaging (DTI) to identify which newborns with Krabbe disease need immediate treatment. Researchers will follow 100 infants with low enzyme levels to see if brain scans can predict which babies will develop the ra…
Matched conditions: KRABBE DISEASE
Sponsor: University of Pittsburgh • Aim: Diagnosis
Last updated Mar 23, 2026 15:15 UTC