Table 2 Examples of computational technologies used for rare disease drug discovery
From: A generalizable pre-clinical research approach for orphan disease therapy
Strategy | Rare disease | Computational Technology | Drug | Reference |
|---|---|---|---|---|
Small Molecule Upregulation | Spinal Muscular Atrophy | Connectivity Map | Anisomycin | [30] |
Small Molecule Upregulation | Spinal Muscular Atrophy | Transcription factor binding site identification | Prolactin | [31] |
Chaperone: drug safety predictions | Gaucher disease | Leadscope | Core structures of aminoquinoline, sulfonamide, and triazine | [71] |
Chaperone: identify binding sites and compounds | Huntington disease | AutoDock, Patch Dock Server, CastP | Metoprolol, minocyclines, and 18 F fluorodeoxyglucose | [72] |
Drug similarity predictions | Neurodegenerative disorders due to protein misfolding | Mode of Action by Network Analysis, MANTRA | Fasudil | [27] |
Prediction of which mutations respond to treatment | Fabry disease | Position specific substitution matrix | 1-deoxy-galactonojirimycin |