Developing a unified QSP platform for lysosomal storage disorders (LSDs)

Systems Pharmacology
Modeling & simulation


Develop a unified modeling platform for LSDs to enhance the comprehension of Lysosomal storage disorders by highlighting differences and common properties between ASMD, Gaucher type 1 and type 3 and Fabry.

What we did

The model was implemented as a set of Ordinary Differential Equations (ODEs) starting from a set of Quantitative System Pharmacology (QSP) models that were specifically developed for each considered disease. The single QSP models have been integrated by an in-depth analysis to obtain a hierarchical unified description that represents each biological process with the most accurate description among the ones developed for the single models.



The platform allows to simulate the four diseases either separately or in parallel. In addition, the platform is equipped with a plot facility that represents simulation results of the disease models in parallel allowing their comparison. Additional modules have been also included to test treatment efficacy in different disease conditions.


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Abrams R, et al. 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 9(7):374-383, 2020.

Kaddi C, et al. Integrated quantitative systems pharmacology (QSP) model of lysosomal diseases provides an innovative computational platform to support research and therapeutic development for the sphingolipidoses. Molecular Genetics and Metabolism 123(2):S73-S74, 2018.

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