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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7455349/
https://www.ncbi.nlm.nih.gov/pubmed/32616675
http://dx.doi.org/10.1212/WNL.0000000000009929

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7455349/
https://www.ncbi.nlm.nih.gov/pubmed/32616675
http://dx.doi.org/10.1212/WNL.0000000000009929

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