Miglustat
| Evidence Level: L5 | Predicted Indications: 51 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Miglustat |
| DrugBank ID | DB00419 |
| Brand Names (EU) | Opfolda |
| Evidence Level | L5 |
| Predicted Indications | 51 |
| Top Prediction Score | 99.88% |
Approved Indication (EMA)
Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA] deficiency).
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | Gaucher disease | 99.88% | DL |
| 2 | autosomal ichthyosis syndrome with fatal disease course | 99.83% | DL |
| 3 | cholesteryl ester storage disease | 99.82% | DL |
| 4 | Krabbe disease | 99.78% | DL |
| 5 | metachromatic leukodystrophy | 99.77% | DL |
| 6 | Wolman disease with hypolipoproteinemia and acanthocytosis | 99.76% | DL |
| 7 | encephalopathy due to prosaposin deficiency | 99.75% | DL |
| 8 | Tay-Sachs disease | 99.75% | DL |
| 9 | benign neoplasm of adrenal gland | 99.74% | DL |
| 10 | recessive X-linked ichthyosis | 99.73% | DL |
| 11 | fatty acid hydroxylase-associated neurodegeneration | 99.72% | DL |
| 12 | lysosomal acid lipase deficiency | 99.70% | DL |
| 13 | cholesterol metabolism disease | 99.69% | DL |
| 14 | adult Krabbe disease | 99.69% | DL |
| 15 | polycystic kidney disease 3 with or without polycystic liver disease | 99.67% | DL |
| 16 | long chain 3-hydroxyacyl-CoA dehydrogenase deficiency | 99.66% | DL |
| 17 | multiple mitochondrial dysfunctions syndrome | 99.63% | DL |
| 18 | cerebrotendinous xanthomatosis | 99.60% | DL |
| 19 | Dorfman-Chanarin disease | 99.60% | DL |
| 20 | fatty acyl-CoA reductase 1 deficiency | 99.58% | DL |
Showing top 20 of 51 predictions.
About TxGNN Predictions
Prediction Sources
| Source | Description |
|---|---|
| KG | Knowledge Graph - Network topology-based associations |
| DL | Deep Learning - Neural network score prediction |
Evidence Levels
| Level | Definition |
|---|---|
| L1 | Multiple Phase 3 RCTs / Systematic Reviews |
| L2 | Single RCT or multiple Phase 2 trials |
| L3 | Observational studies / Large case series |
| L4 | Preclinical / Mechanistic / Case reports |
| L5 | AI prediction only (current) |
Clinical Validation Needed
Research Use Only: These predictions are computational hypotheses that require clinical validation. They should NOT be used for clinical decision-making.
Next Steps for Validation
- Literature Review: Search PubMed for existing evidence
- Clinical Trial Search: Check ClinicalTrials.gov for ongoing studies
- Mechanistic Analysis: Evaluate biological plausibility
- Preclinical Studies: Conduct in vitro/in vivo validation
- Clinical Trials: Design and conduct human studies
Data Access
- FHIR API:
/fhir/ClinicalUseDefinition/ - CSV Download: All Predictions
- GitHub: yao-care/EuTxGNN
Citation
If using this data, please cite:
@article{huang2023txgnn,
title={A foundation model for clinician-centered drug repurposing},
author={Huang, Kexin and others},
journal={Nature Medicine},
year={2023},
doi={10.1038/s41591-023-02233-x}
}
Disclaimer: This report is for research purposes only and does not constitute medical advice. Drug repurposing predictions require rigorous clinical validation before any therapeutic application.