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

• Κύριοι συγγραφείς: Gabrielle Ghabash (Συγγραφέας), Jacob Wilkes (Συγγραφέας), Joshua L. Bonkowsky (Συγγραφέας)
• Μορφή: Βιβλίο
• Έκδοση: Frontiers Media S.A., 2021-11-01T00:00:00Z.
• Θέματα: article
• Διαθέσιμο Online: Connect to this object online.

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...