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