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Neurodegenerative disease after hematopoietic stem cell transplantation in metachromatic leukodystrophy

OBJECTIVE: Metachromatic leukodystrophy is a lysosomal storage disease caused by deficient arylsulfatase A. It is characterized by progressive demyelination and thus mainly affects the white matter. Hematopoietic stem cell transplantation may stabilize and improve white matter damage, yet some patie...

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Detalles Bibliográficos
Autores principales: Al‐Saady, Murtadha, Beerepoot, Shanice, Plug, Bonnie C., Breur, Marjolein, Galabova, Hristina, Pouwels, Petra J. W., Boelens, Jaap‐Jan, Lindemans, Caroline, van Hasselt, Peter M., Matzner, Ulrich, Vanderver, Adeline, Bugiani, Marianna, van der Knaap, Marjo S., Wolf, Nicole I.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10351661/
https://www.ncbi.nlm.nih.gov/pubmed/37212343
http://dx.doi.org/10.1002/acn3.51796