Rare disease · Lysosomal storage
ASMD / Xenpozyme QSP v2.0
Adult and paediatric olipudase alfa ERT model for acid sphingomyelinase deficiency.
Model description
Adult and paediatric olipudase alfa ERT model for acid sphingomyelinase deficiency.
What can be done with this model
- Residual ASM activity and sphingomyelin burden
- Olipudase alfa PK, tissue uptake and lysosomal delivery
- Lyso‑SM, ceramide, spleen, liver, DLCO and platelet endpoints
- Adult-to-paediatric dose-bridging and regimen optimisation
Free/open layer
- SBML v2.0 adult and paediatric files
- v0.2/v0.3 development SBML scaffold
- Selected CSV/JSON reports
- Figures and static artefact bundle
Subscription layer
- Quarterly re-calibration against new ASCEND/OLE evidence
- Patient-level virtual twin extensions
- Regulatory V&V40 dossier and COU refinement
- Partner-specific dose and benefit-risk scenarios
ASMD v2.0 pipeline outputs
11species per population SBML
53parameters per population SBML
27dose events
96.0%calibration PASS rate
Downloads
Adult SBML v2.0 downloadPaediatric SBML v2.0 downloadFull ASMD v2 bundle downloadDecision table CSV downloadModel summary JSON downloadPredictions CSV calibration stageV&V report JSON validationMaster pipeline summary Markdownv0.2 calibration workbook XLSX
The v0.2 scaffold files are retained as development artefacts. The v2.0 adult and paediatric SBML files are the current ASMD model assets in this website package.