ArticleCPT: pharmacometrics & systems pharmacology2018
A Semi-Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy.
Article in CPT: pharmacometrics & systems pharmacology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
16 citing papers in PubMed, 27 citations in OpenAlex.
- Physiologically-Based Pharmacokinetic Modeling to Support Pediatric Clinical Development: An IQ Working Group Perspective on the Current Status and Challenges.CPT: pharmacometrics & systems pharmacology · 2026Review
- Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence.The Journal of international medical research · 2025Review
- Translational and clinical development of therapeutic siRNA and ASOs: current industry practices, perspectives, and recommendations.Nucleic acids research · 2025Article
- Tissue distribution pharmacokinetics of intrathecal U1 adaptor oligonucleotide in mice.Current research in pharmacology and drug discovery · 2025Article
- OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides.Nucleic acid therapeutics · 2023Article
- Development of a population pharmacokinetic model to characterize the pharmacokinetics of intrathecally administered tominersen in cerebrospinal fluid and plasma.CPT: pharmacometrics & systems pharmacology · 2023Article
- A pediatric quantitative systems pharmacology model of neurofilament trafficking in spinal muscular atrophy treated with the antisense oligonucleotide nusinersen.CPT: pharmacometrics & systems pharmacology · 2023Article
- A mechanistic pharmacokinetic model for intrathecal administration of antisense oligonucleotides.Frontiers in physiology · 2023Article
- Applications, Challenges, and Outlook for PBPK Modeling and Simulation: A Regulatory, Industrial and Academic Perspective.Pharmaceutical research · 2022Review
- Translational Approaches for Brain Delivery of Biologics via Cerebrospinal Fluid.Clinical pharmacology and therapeutics · 2022Review
- A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration.Journal of pharmacokinetics and pharmacodynamics · 2021Article
- Minimally Invasive Nasal Depot (MIND) technique for direct BDNF AntagoNAT delivery to the brain.Journal of controlled release : official journal of the Controlled Release Society · 2021Article
- Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- The Antisense Oligonucleotide Nusinersen for Treatment of Spinal Muscular Atrophy.Orthopedic reviews · 2021Review
- Pharmacokinetic Modeling of the Impact of P-glycoprotein on Ondansetron Disposition in the Central Nervous System.Pharmaceutical research · 2020Article
- Targeting RNA: A Transformative Therapeutic Strategy.Clinical and translational science · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A pharmacokinetic (PK) model was developed for nusinersen, an antisense oligonucleotide (ASO) that is the first approved treatment for spinal muscular atrophy (SMA). The model was built with data from 92 nonhuman primates (NHPs) following intrathecal doses (0.3-7 mg) and characterized the PK in cerebrospinal fluid (CSF), plasma, total spinal cord, brain, and pons. The estimated volumes were 13.6, 937, 4.5, 53.8, and 2.11 mL, respectively. Global sensitivity analysis demonstrated that the CSF-to-plasma drug distribution rate (0.09 hour
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.