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Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series

This longitudinal cohort study aimed to determine whether circulating neurofilaments (NFs) can monitor response to molecular therapies in newborns with spinal muscular atrophy (SMA; NCT02831296). We applied a mixed-effect model to examine differences in serum NF levels among healthy control infants...

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Detalles Bibliográficos
Autores principales: Alves, Christiano R.R., Petrillo, Marco, Spellman, Rebecca, Garner, Reid, Zhang, Ren, Kiefer, Michael, Simeone, Sarah, Sohn, Jihee, Eichelberger, Eric J., Rodrigues, Emma, Arruda, Elizabeth A., Townsend, Elise L., Farwell, Wildon, Swoboda, Kathryn J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8605296/
https://www.ncbi.nlm.nih.gov/pubmed/34853799
http://dx.doi.org/10.1016/j.omtm.2021.10.011