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Article

Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy.

FASEB journal : official publication of the Federation of American Societies for Experimental Biology - 1 Sept 2005

Lee Wing C, Courtenay Andrew, Troendle Frederick J, Stallings-Mann Melody L, Dickey Chad A, DeLucia Michael W, Dickson Dennis W, Eckman Christopher B

Abstract excerpt

Globoid cell leukodystrophy (GLD) or Krabbe disease is a devastating, degenerative neurological disorder caused by mutations in the galactosylceramidase (GALC) gene that severely affect enzyme activity. Currently, treatment options for this disorder are very limited. Enzyme replacement therapy (ERT) has been shown to be effective in lysosomal storage disorders with predominantly peripheral manifestations such as...

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Source

Identifiers

PMID: 15987783
DOI: 10.1096/fj.05-3826fje

Genes

GALC

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy.PMID 15987783Read ↗

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