Carglumic Acid
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Carglumic Acid |
| DrugBank ID | DB06775 |
| Brand Names (EU) | Ucedane |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 86.61% |
Approved Indication (EMA)
Ucedane is indicated in treatment of: hyperammonaemia due to N-acetylglutamate synthase primary deficiency; Hyperammonaemia due to isovaleric acidaemia; Hyperammonaemia due to methymalonic acidaemia; Hyperammonaemia due to propionic acidaemia.
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | cystic teratoma | 86.61% | DL |
| 2 | spinal cord dermoid cyst | 86.47% | DL |
| 3 | disease of orbital part of eye adnexa | 86.27% | DL |
| 4 | disease of orbital region | 86.06% | DL |
| 5 | dermoid cyst of ovary | 85.85% | DL |
| 6 | eye disease | 83.64% | DL |
| 7 | open-angle glaucoma | 83.33% | DL |
| 8 | primary hereditary glaucoma | 83.22% | DL |
| 9 | chronic relapsing inflammatory optic neuropathy | 83.21% | DL |
| 10 | isolated optic neuritis | 83.21% | DL |
| 11 | recurrent idiopathic neuroretinitis | 83.21% | DL |
| 12 | optic perineuritis | 83.21% | DL |
| 13 | conjunctival degeneration | 83.15% | DL |
| 14 | vitreous detachment | 83.14% | DL |
| 15 | total internal ophthalmoplegia | 83.12% | DL |
| 16 | cycloplegia | 83.12% | DL |
| 17 | accommodative spasm | 83.12% | DL |
| 18 | luxation of globe | 83.06% | DL |
| 19 | microcornea-corectopia-macular hypoplasia syndrome | 83.05% | DL |
| 20 | blepharophimosis-radioulnar synostosis syndrome | 83.05% | 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.