Nitisinone
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Nitisinone |
| DrugBank ID | DB00348 |
| Brand Names (EU) | Nityr |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 99.96% |
Approved Indication (EMA)
Treatment of adult and paediatric patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | renal tubular acidosis | 99.96% | DL |
| 2 | galactosemia | 99.95% | DL |
| 3 | serpinopathy with toxic serpin polymerization | 99.90% | DL |
| 4 | C1 inhibitor deficiency | 99.90% | DL |
| 5 | glycogen storage disease | 99.89% | DL |
| 6 | adult polyglucosan body disease | 99.88% | DL |
| 7 | glycogen storage disease due to glucose-6-phosphatase deficiency | 99.87% | DL |
| 8 | Griscelli syndrome | 99.87% | DL |
| 9 | granulomatous disease, chronic, X-linked | 99.87% | DL |
| 10 | ermine phenotype | 99.87% | DL |
| 11 | ocular albinism (disease) | 99.86% | DL |
| 12 | piebaldism | 99.86% | DL |
| 13 | ocular albinism with sensorineural deafness | 99.86% | DL |
| 14 | anemia, nonspherocytic hemolytic, due to G6PD deficiency | 99.85% | DL |
| 15 | cold agglutinin disease | 99.85% | DL |
| 16 | primary CD59 deficiency | 99.85% | DL |
| 17 | hemolytic anemia due to diphosphoglycerate mutase deficiency | 99.85% | DL |
| 18 | cholelithiasis | 99.84% | DL |
| 19 | albinism | 99.84% | DL |
| 20 | classic galactosemia | 99.84% | 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.