Development and validation of the Charcot-Marie-Tooth Disease Infant Scale

Many genetic subtypes of Charcot-Marie-Tooth disease (CMT) show signs of symptomatic disease during the earliest years of life. This might be the ideal time to intervene before progression of clinical sequelae due to demyelination and axonal loss. In the absence of disease-specific clinical trial ou...

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Gepubliceerd in:Brain
Hoofdauteurs: Mandarakas, Melissa R, Menezes, Manoj P, Rose, Kristy J, Shy, Rosemary, Eichinger, Kate, Foscan, Maria, Estilow, Timothy, Kennedy, Rachel, Herbert, Karen, Bray, Paula, Refshauge, Kathryn, Ryan, Monique M, Yiu, Eppie M, Farrar, Michelle, Sampaio, Hugo, Moroni, Isabella, Pagliano, Emanuela, Pareyson, Davide, Yum, Sabrina W, Herrmann, David N, Acsadi, Gyula, Shy, Michael E, Burns, Joshua, Sanmaneechai, Oranee
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2018
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Original Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6312041/
https://ncbi.nlm.nih.gov/pubmed/30476010
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/brain/awy280
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