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Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis
(2022-03)
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative
treatment for patients affected by Wiskott-Aldrich syndrome (WAS). Reported HSCT
outcomes have improved over time with respect to overall ...