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Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy

IMPORTANCE: Allogeneic hematopoietic stem cell transplantation is the standard intervention for childhood cerebral X-linked adrenoleukodystrophy. However, the pretransplant conditions, demyelination patterns, complications, and neurological outcomes of this therapy are not well characterized. OBJECT...

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Detalles Bibliográficos
Autores principales: Kühl, Jörn-Sven, Kupper, Jana, Baqué, Hermann, Ebell, Wolfram, Gärtner, Jutta, Korenke, Christoph, Spors, Birgit, Steffen, Ingo G., Strauss, Gabriele, Voigt, Sebastian, Weschke, Bernhard, Weddige, Almuth, Köhler, Wolfgang, Steinfeld, Robert
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Medical Association 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6324299/
https://www.ncbi.nlm.nih.gov/pubmed/30646031
http://dx.doi.org/10.1001/jamanetworkopen.2018.0769