Alglucosidase Alfa
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Alglucosidase Alfa |
| DrugBank ID | DB01272 |
| Brand Names (EU) | Myozyme |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 99.47% |
Approved Indication (EMA)
Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid-?-glucosidase deficiency). In patients with late-onset Pompe disease the evidence of efficacy is limited.
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | adult polyglucosan body disease | 99.47% | DL |
| 2 | glycogen storage disease due to glycogen branching enzyme deficiency, congenital neuromuscular form | 99.32% | DL |
| 3 | glycogen storage disease due to glycogen branching enzyme deficiency, fatal perinatal neuromuscular form | 99.32% | DL |
| 4 | congenital entropion | 99.22% | DL |
| 5 | congenital ectropion | 99.17% | DL |
| 6 | congenital Horner syndrome (disease) | 99.17% | DL |
| 7 | ptosis-vocal cord paralysis syndrome | 99.15% | DL |
| 8 | camptodactyly, myopia, and fibrosis of the medial rectus muscle of eye | 99.11% | DL |
| 9 | epiblepharon | 99.11% | DL |
| 10 | ptosis-strabismus-ectopic pupils syndrome | 99.10% | DL |
| 11 | tricarboxylic acid cycle disorder | 99.07% | DL |
| 12 | ptosis-upper ocular movement limitation-absence of lacrimal punctum syndrome | 99.01% | DL |
| 13 | mucopolysaccharidosis | 98.97% | DL |
| 14 | jaw-winking syndrome | 98.97% | DL |
| 15 | disease of transporter activity | 98.97% | DL |
| 16 | glycogen storage disease | 98.92% | DL |
| 17 | glycogen storage disease due to glycogen branching enzyme deficiency, adult neuromuscular form | 98.81% | DL |
| 18 | glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form | 98.81% | DL |
| 19 | glycogen storage disease due to glycogen branching enzyme deficiency, childhood combined hepatic and myopathic form | 98.81% | DL |
| 20 | glycogen storage disease due to glycogen branching enzyme deficiency, progressive hepatic form | 98.81% | 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.