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

• Main Authors: Alessio Paris (Author), Pranami Bora (Author), Silvia Parolo (Author), Drew MacCannell (Author), Michael Monine (Author), Nick van derMunnik (Author), Xiao Tong (Author), Satish Eraly (Author), Zdenek Berger (Author), Danielle Graham (Author), Toby Ferguson (Author), Enrico Domenici (Author), Ivan Nestorov (Author), Luca Marchetti (Author)
• Format: Book
• Published: Wiley, 2023-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...