ArticleCPT: pharmacometrics & systems pharmacology2018
Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology.
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 21 papers.
What it found
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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
21 citing papers in PubMed.
- From Biological Mechanisms to Causal Inference: Quantitative Systems Pharmacology and Causal Frameworks in Drug Development.Clinical pharmacology and therapeutics · 2026Article
- From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development.Journal of clinical pharmacology · 2026Review
- Model-Informed Drug Development: Addressing the Critical Need for Training in the Promising New Field.Delaware journal of public health · 2026Article
- The Impact of QSP Modeling on the Design and Optimization of Gene Therapy Approaches.CPT: pharmacometrics & systems pharmacology · 2025Review
- Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists.Clinical and translational science · 2025Review
- The Potential of Disease Progression Modeling to Advance Clinical Development and Decision Making.Clinical pharmacology and therapeutics · 2025Review
- Application of model-informed drug development (MIDD) for dose selection in regulatory submissions for drug approval in Japan.Journal of pharmacokinetics and pharmacodynamics · 2025Article
- Application of Quantitative Systems Pharmacology Approaches to Support Pediatric Labeling in Rare Diseases.Handbook of experimental pharmacology · 2025Review
- Landscape of regulatory quantitative systems pharmacology submissions to the U.S. Food and Drug Administration: An update report.CPT: pharmacometrics & systems pharmacology · 2024Article
- Creating a Roadmap to Quantitative Systems Pharmacology-Informed Rare Disease Drug Development: A Workshop Report.Clinical pharmacology and therapeutics · 2024Review
- Advancing precision medicine therapeutics for Parkinson's utilizing a shared quantitative systems pharmacology model and framework.Frontiers in systems biology · 2024Article
- Achieving big with small: quantitative clinical pharmacology tools for drug development in pediatric rare diseases.Journal of pharmacokinetics and pharmacodynamics · 2023Review
- Role of Disease Progression Models in Drug Development.Pharmaceutical research · 2022Review
- QSPcc reduces bottlenecks in computational model simulations.Communications biology · 2021Article
- Reconstruction of the Cytokine Signaling in Lysosomal Storage Diseases by Literature Mining and Network Analysis.Frontiers in cell and developmental biology · 2021Article
- History and Future Perspectives on the Discipline of Quantitative Systems Pharmacology Modeling and Its Applications.Frontiers in physiology · 2021Review
- A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat.CPT: pharmacometrics & systems pharmacology · 2020Article
- Translational Quantitative Systems Pharmacology in Drug Development: from Current Landscape to Good Practices.The AAPS journal · 2019Review
- Industrialization of Quantitative Systems Pharmacology.CPT: pharmacometrics & systems pharmacology · 2019Article
- Reproducibility of Quantitative Systems Pharmacology Models: Current Challenges and Future Opportunities.CPT: pharmacometrics & systems pharmacology · 2019Review
Corrections and comments
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Authors and funding
16 authors.
Funding
Abstract
Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder with heterogeneous clinical manifestations, including hepatosplenomegaly and infiltrative pulmonary disease, and is associated with significant morbidity and mortality. Olipudase alfa (recombinant human acid sphingomyelinase) is an enzyme replacement therapy under development for the non-neurological manifestations of ASMD. We present a quantitative systems pharmacology (QSP) model supporting the clinical development of olipudase alfa. The model is multiscale and mechanistic, linking the enzymatic deficiency driving the disease to molecular-level, cellular-level, and organ-level effects. Model development was informed by natural history, and preclinical and clinical studies. By considering patient-specific pharmacokinetic (PK) profiles and indicators of disease severity, the model describes pharmacodynamic (PD) and clinical end points for individual patients. The ASMD QSP model provides a platform for quantitatively assessing systemic pharmacological effects in adult and pediatric patients, and explaining variability within and across these patient populations, thereby supporting the extrapolation of treatment response from adults to pediatrics.
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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 Socratic graph.