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Infantile Krabbe disease
MONDO:0016089Also known as: Krabbe disease, classic form, Krabbe disease, early-onset, early onset Krabbe disease, early symptomatic Krabbe disease, early-onset Krabbe disease, infantile onset Krabbe disease, infantile-onset Krabbe disease
8 clinical trials for this condition and its sub-types, 2 tagged with Infantile Krabbe disease itself.
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Experimental gene therapy aims to halt devastating brain disease in infants
Disease control PausedThis study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best…
Phase 1/2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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Gene therapy after stem cell transplant shows promise for rare brain disease
Disease control OngoingThis study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gen…
Phase 1/2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC