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Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation

Hurler syndrome (HS) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Hematopoietic stem cell transplantation (HSCT) results in long-term survival, although with significant residual disease burden. How this residual disease affects the health-relat...

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Bibliografiske detaljer
Udgivet i:Blood Adv
Main Authors: Aldenhoven, Mieke, van den Broek, Brigitte T. A., Wynn, Robert F., O’Meara, Anne, Veys, Paul, Rovelli, Attilio, Jones, Simon A., Parini, Rossella, van Hasselt, Peter M., Renard, Marleen, Bordon, Victoria, de Koning, Tom J., Boelens, Jaap Jan
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Hematology 2017
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5737136/
https://ncbi.nlm.nih.gov/pubmed/29296871
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2017011387
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