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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Format: | Book |
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Frontiers Media S.A.,
2021-11-01T00:00:00Z.
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A1234.567 |
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