Table 2.

Examples of computational technologies used for rare disease drug discovery

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]