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

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 ...

Source: Orphanet Journal of Rare Diseases - February 18, 2019 Category: Internal Medicine Authors: Maria L. Beltran-Quintero, Nicholas A. Bascou, Michele D. Poe, David A. Wenger, Carlos A. Saavedra-Matiz, Matthew J. Nichols and Maria L. Escolar Tags: Research Source Type: research