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

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

Abstract 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 ag...

Full description

Bibliographic Details
Main Authors: Alessio Paris, Pranami Bora, Silvia Parolo, Drew MacCannell, Michael Monine, Nick van derMunnik, Xiao Tong, Satish Eraly, Zdenek Berger, Danielle Graham, Toby Ferguson, Enrico Domenici, Ivan Nestorov, Luca Marchetti
Format: Article
Language:English
Published: Wiley 2023-02-01
Series:CPT: Pharmacometrics & Systems Pharmacology
Online Access:https://doi.org/10.1002/psp4.12890

Internet

https://doi.org/10.1002/psp4.12890

Similar Items