First hematopoietic stem cell transplantation in a child with late infantile Krabbe disease in Poland
Krabbe disease (KD) is a lysosomal storage disorder, caused by a deficiency of galactocerebrosidase (GALC) or a mutation in the prosaposin gene, which leads to psychosine accumulation. Consequently, demyelination and degradation of the nervous system occur. Newborns with early infantile Krabbe disea...
Main Authors: | , , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
Termedia Publishing House
2021-03-01
|
Series: | Pediatria Polska |
Subjects: | |
Online Access: | https://www.termedia.pl/First-hematopoietic-stem-cell-transplantation-in-a-child-with-late-infantile-Krabbe-disease-in-Poland,127,43674,1,1.html |