Nusinersen Sodium

Evidence Level: L5 Predicted Indications: 50

Quick Overview

Item Value
Drug Name Nusinersen Sodium
DrugBank ID DB13161
Brand Names (EU) Spinraza
Evidence Level L5
Predicted Indications 50
Top Prediction Score 50.00%

Approved Indication (EMA)

Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.


Predicted New Indications

TxGNN model predictions for potential drug repurposing:

Rank Indication Score Source
1 PRPS1 deficiency disorder 50.00% DL
2 hypervalinemia and hyperleucine-isoleucinemia 50.00% DL
3 exercise-induced anaphylaxis 50.00% DL
4 acetazolamide-responsive hereditary episodic ataxia 50.00% DL
5 cerebral folate deficiency 50.00% DL
6 adolescent/adult onset autosomal dominant epilepsy with auditory features 50.00% DL
7 antibody mediated epilepsy 50.00% DL
8 febrile seizures plus, genetic epilepsy with febrile seizures plus 50.00% DL
9 food protein-induced allergic proctocolitis 50.00% DL
10 MED12-related intellectual disability syndrome 50.00% DL
11 rod-cone dystrophy, sensorineural deafness, and Fanconi-type renal dysfunction 50.00% DL
12 mixed mineral dust pneumoconiosis 50.00% DL
13 epilepsy of infancy with migrating focal seizures 50.00% DL
14 neonatal/infantile epilepsy syndrome 50.00% DL
15 primary mast cell activation syndrome 50.00% DL
16 adult-onset segmental dystonia 50.00% DL
17 paratenonitis with tendinosis 50.00% DL
18 tendinosis 50.00% DL
19 TH-deficient infantile parkinsonism and motor delay 50.00% DL
20 saccharopinuria 50.00% 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

  1. Literature Review: Search PubMed for existing evidence
  2. Clinical Trial Search: Check ClinicalTrials.gov for ongoing studies
  3. Mechanistic Analysis: Evaluate biological plausibility
  4. Preclinical Studies: Conduct in vitro/in vivo validation
  5. Clinical Trials: Design and conduct human studies

Data Access


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.

Copyright © 2026 EuTxGNN Project. For research purposes only. Not medical advice.