Krabbe disease

MONDO:0009499

A 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

21 clinical trials for this condition and its sub-types, 15 tagged with Krabbe disease itself.

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Where it sits in the disease tree

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Part of

↑ Hereditary peripheral neuropathy (479) ↑ Sphingolipidosis (166) ↑ Eye degenerative disorder (97) ↑ Leukodystrophy (72)

Sub-types of Krabbe disease

Infantile Krabbe disease 2 trials
Adult Krabbe disease 0 trials
Late-infantile/juvenile Krabbe disease 0 trials

Including sub-types (21) Tagged with Krabbe disease (15)

Trials to join now! 6 Not yet recruiting 1 Not yet finished but already full! 6 Completed 1 Terminated 1

Gene therapy for krabbe disease: did it last?

Knowledge-focused Stopped early

This 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

Krabbe disease

Where it sits in the disease tree

Gene therapy for krabbe disease: did it last?