Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months

Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months

Abstract Background Krabbe disease is a rare neurological disorder caused by a deficiency in the lysosomal enzyme, β-galactocerebrosidase, resulting in demyelination of the central and peripheral nervous systems. If left without treatment, Krabbe disease results in progressive neurodegeneration with...

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Bibliographic Details
Main Authors: Maria L. Beltran-Quintero, Nicholas A. Bascou, Michele D. Poe, David A. Wenger, Carlos A. Saavedra-Matiz, Matthew J. Nichols, Maria L. Escolar
Format: Article
Language:English
Published: BMC 2019-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Krabbe disease
Globoid cell leukodystrophy
Early-infantile
Infantile
Natural history
Newborn screening
Online Access:http://link.springer.com/article/10.1186/s13023-019-1018-4

Internet

http://link.springer.com/article/10.1186/s13023-019-1018-4

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