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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Bibliographic Details
Main Authors: Gabrielle Ghabash (Author), Jacob Wilkes (Author), Joshua L. Bonkowsky (Author)
Format: Book
Published: Frontiers Media S.A., 2021-11-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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