Laronidase
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Laronidase |
| DrugBank ID | DB00090 |
| Brand Names (EU) | Aldurazyme |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 99.74% |
Approved Indication (EMA)
Aldurazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis I (MPS I; alpha-L-iduronidase deficiency) to treat the nonneurological manifestations of the disease.
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | Scheie syndrome | 99.74% | DL |
| 2 | lysosomal storage disease with skeletal involvement | 99.31% | DL |
| 3 | Hurler syndrome | 99.23% | DL |
| 4 | Sanfilippo syndrome | 99.22% | DL |
| 5 | lysosomal disease with hypertrophic cardiomyopathy | 98.82% | DL |
| 6 | syndromic neurometabolic disease with X-linked intellectual disability | 98.73% | DL |
| 7 | eyelids malposition disorder | 98.65% | DL |
| 8 | camptodactyly, myopia, and fibrosis of the medial rectus muscle of eye | 98.63% | DL |
| 9 | ptosis-strabismus-ectopic pupils syndrome | 98.57% | DL |
| 10 | inborn disorder of lysosomal amino acid transport | 98.56% | DL |
| 11 | ptosis-upper ocular movement limitation-absence of lacrimal punctum syndrome | 98.56% | DL |
| 12 | ptosis-vocal cord paralysis syndrome | 98.55% | DL |
| 13 | congenital Horner syndrome (disease) | 98.50% | DL |
| 14 | Steel syndrome | 98.48% | DL |
| 15 | Hurler-Scheie syndrome | 98.42% | DL |
| 16 | jaw-winking syndrome | 98.37% | DL |
| 17 | congenital entropion | 98.29% | DL |
| 18 | epiblepharon | 98.21% | DL |
| 19 | mucopolysaccharidosis | 98.20% | DL |
| 20 | congenital ectropion | 98.17% | DL |
Showing top 20 of 50 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.