Krabbe disease
MONDO:0009499A lysosomal disorder that affects the white matter of the central and peripheral nervous systems. It includes infantile, late-infantile/juvenile and adult forms.
Also known as: GALC deficiency, GALC enzyme deficiency, Krabbe disease, Krabbe's leukodystrophy, Leukodystrophy, Krabbe's, diffuse globoid body sclerosis, galactocerebrosidase deficiency, galactosylceramidase deficiency
22 clinical trials for this condition and its sub-types.
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Broader categories
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Could a simple blood test track nerve damage? a study investigates NT-3 levels in neuropathy.
Knowledge-focused TerminatedThis study measures levels of a protein called NT-3 in the blood of people with peripheral neuropathy or Charcot-Marie-Tooth disease. Researchers will compare these levels with measures of muscle strength, mobility, and daily function. The goal is to see whether NT-3 levels corre…
Sponsor: Zarife Sahenk • Aim: Knowledge-focused
Last updated Jul 31, 2026 00:00 UTC
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Gene therapy for krabbe disease: did it last?
Knowledge-focused TerminatedThis study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.
Sponsor: Forge Biologics, Inc • Aim: Knowledge-focused
Last updated Jun 26, 2026 13:47 UTC