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Neurofilament light is a treatment‐responsive biomarker in CLN2 disease

OBJECTIVE: Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, progressive, fatal neurodegenerative pediatric disorder resulting from deficiencies of the lysosomal enzyme tripeptidyl peptidase 1 that are caused by mutations in TPP1. Identifying biomarkers of CLN2 disease progression will...

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Detalles Bibliográficos
Autores principales: Ru, Yuanbin, Corado, Carley, Soon, Russell K., Melton, Andrew C., Harris, Adam, Yu, Guoying K., Pryer, Nancy, Sinclair, John R., Katz, Martin L., Ajayi, Temitayo, Jacoby, David, Russell, Chris B., Chandriani, Sanjay
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6917340/
https://www.ncbi.nlm.nih.gov/pubmed/31814335
http://dx.doi.org/10.1002/acn3.50942