Hurdles in treating Hurler disease: potential routes to achieve a “real” cure

Mucopolysaccharidoses (MPSs) are multiorgan devastating diseases for which hematopoietic cell transplantation (HCT) and, to a lesser extent, enzyme replacement therapy have substantially altered the course of the disease. Furthermore, they have resulted in increased overall survival, especially for...

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Veröffentlicht in:Blood Adv
Hauptverfasser: van den Broek, Brigitte T. A., van Doorn, Jaap, Hegeman, Charlotte V., Nierkens, Stefan, Lindemans, Caroline A., Verhoeven-Duif, Nanda, Boelens, Jaap Jan, van Hasselt, Peter M.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Hematology 2020
Schlagworte:
Review Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7322951/
https://ncbi.nlm.nih.gov/pubmed/32574368
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020001708
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