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...
Guardado en:
Autores principales: | Gabrielle Ghabash, Jacob Wilkes, Joshua L. Bonkowsky |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Frontiers Media S.A.
2021
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Materias: | |
Acceso en línea: | https://doaj.org/article/b015126da5714d1aad426af2846253cf |
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