MRI surveillance of boys with X-linked adrenoleukodystrophy identified by newborn screening: Meta-analysis and consensus guidelines

BACKGROUND: Among boys with X-Linked adrenoleukodystrophy, a subset will develop childhood cerebral adrenoleukodystrophy (CCALD). CCALD is typically lethal without hematopoietic stem cell transplant before or soon after symptom onset. We sought to establish evidence-based guidelines detailing the ne...

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Udgivet i:J Inherit Metab Dis
Main Authors: Mallack, Eric J., Turk, Bela R., Yan, Helena, Price, Carrie, Demetres, Michelle, Moser, Ann B., Becker, Catherine, Hollandsworth, Kim, Adang, Laura, Vanderver, Adeline, Van Haren, Keith, Ruzhnikov, Maura, Kurtzberg, Joanne, Maegawa, Gustavo, Orchard, Paul J., Lund, Troy C., Raymond, Gerald V., Regelmann, Molly, Orsini, Joseph J., Seeger, Elisa, Kemp, Stephan, Eichler, Florian, Fatemi, Ali
Format: Artigo
Sprog:Inglês
Udgivet: 2021
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8113077/
https://ncbi.nlm.nih.gov/pubmed/33373467
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12356
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