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Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy

OBJECTIVE: To quantify benchmark treatment outcomes that may be enabled by newborn screening surveillance for X-linked adrenoleukodystrophy (ALD), we report neurocognitive, neuropsychiatric, and MRI change for boys who underwent hematopoietic stem cell transplant (HSCT) at initial stages of demyelin...

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Detalhes bibliográficos
Publicado no:Neurology
Main Authors: Pierpont, Elizabeth I., Nascene, David R., Shanley, Ryan, Kenney-Jung, Daniel L., Ziegler, Richard S., Miller, Weston P., Gupta, Ashish O., Lund, Troy C., Orchard, Paul J., Eisengart, Julie B.
Formato: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7455349/
https://ncbi.nlm.nih.gov/pubmed/32616675
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000009929
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