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National U.S. Patient and Transplant Data for Krabbe Disease

Krabbe disease (KD) is a leukodystrophy caused by mutations in the galactosylceramidase gene. Presymptomatic hematopoietic stem cell transplantation (HSCT) is associated with improved outcomes, but most data are from single-center studies. Our objective was to characterize national patterns of HSCT...

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Detalles Bibliográficos
Autores principales: Ghabash, Gabrielle, Wilkes, Jacob, Bonkowsky, Joshua L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8660087/
https://www.ncbi.nlm.nih.gov/pubmed/34900869
http://dx.doi.org/10.3389/fped.2021.764626