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    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 by 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

    Published 2023
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