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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 [73, 74]