A pediatric quantitative systems pharmacology model of neurofilament trafficking in spinal muscular atrophy treated with the antisense oligonucleotide nusinersen

Phosphorylated neurofilament heavy subunit (pNfH) has been recently identified as a promising biomarker of disease onset and treatment efficacy in spinal muscular atrophy (SMA). This study introduces a quantitative systems pharmacology model representing the SMA pediatric scenario in the age range o...

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Detalles Bibliográficos
Autores principales: Paris, Alessio, Bora, Pranami, Parolo, Silvia, MacCannell, Drew, Monine, Michael, van der Munnik, Nick, Tong, Xiao, Eraly, Satish, Berger, Zdenek, Graham, Danielle, Ferguson, Toby, Domenici, Enrico, Nestorov, Ivan, Marchetti, Luca
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9931427/
https://www.ncbi.nlm.nih.gov/pubmed/36471456
http://dx.doi.org/10.1002/psp4.12890

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9931427/
https://www.ncbi.nlm.nih.gov/pubmed/36471456
http://dx.doi.org/10.1002/psp4.12890

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