DDrare: Database of Drug Development for Rare Diseases

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No.	DrugBank	Drug (Description in trials)	KEGG DRUG	KEGG GENES	KEGG PATHWAY On map, Yellow: Drug target genes	Disease ID
1	Bleomycin	[3] Bleomycin; Bleomycin + fibrovein; Bleomycin baxter;	[3] D02177 D03229 D07535 💬	-	-	[4] 96, 278, 279, 280 💬
2	Indocyanine green	[3] Indocyanine green; Indocyanine green(icg); Verde de indocianina (indocyanine green);	[1] D01342 💬	-	-	[7] 46, 51, 96, 235, 278, 291, 296 💬
3	Propranolol	[5] Atenolol or propranolol; Losartan and atenolol or propranolol; Propranolol; Propranolol hcl cf 40 mg tablet; Propranolol treatment;	[2] D00483 D08443 💬	[3] ADRB1, ADRB2, ADRB3 💬	[11] Adrenergic signaling in cardiomyocytes, Calcium signaling pathway, Dilated cardiomyopathy, Gap junction, Neuroactive ligand-receptor interaction, Regulation of lipolysis in adipocytes, Renin secretion, Salivary secretion, Thermogenesis, cAMP signaling pathway, cGMP-PKG signaling pathway	[6] 6 , 158, 167, 227, 278, 296 💬
4	Selenium	[2] Selenium; Vitamin e/selenium;	-	-	-	[3] 13, 278, 298 💬
5	Sildenafil	[32] 20 mg sildenafil citrate by mouth; 5% sildenafil cream; Access program - sildenafil citrate, viagra, revatio; Bosentan and sildenafil; Duo-therapy with sildenafil + bosentan; Era and pde-5i (sildenafil, tadalafil, bosentan, macitentan); G04be03- sildenafil - sildenafil (citrato); Mono-therapy with sildenafil; Oral sildenafil; Other: standard of care sildenafil; Sildenafil; Sildenafil (50 mg); Sildenafil (acute-1 hour); Sildenafil (as citrate); Sildenafil (revatio); Sildenafil (revatio) 20 mg tid; Sildenafil (subchronic-4 weeks); Sildenafil 20 mg tablets; Sildenafil 20mg; Sildenafil 20mg and bosentan 62.5mg; Sildenafil 40mg oral capsule; Sildenafil and losartan; Sildenafil by injection; Sildenafil citrate; Sildenafil citrate (uk-92,480); Sildenafil filmtabletten; Sildenafil singly or in association with bosentan; Sildenafil therapy; Sildenafilo; Sitaxsentan and sildenafil; Sodium tanshinone iia sulfonate diluted with 5% glucose solution,20mg sildenafil citrate by mouth; Tadalafil and sildenafil;	[2] D02229 D08514 💬	[1] PDE5A 💬	[3] Metabolic pathways, Purine metabolism, cGMP-PKG signaling pathway	[17] 6 , 46, 50, 51, 85, 86, 90, 113, 210, 211, 212, 225, 226, 278, 294, 299, 301 💬
6	Sirolimus	[22] Rapamycin, sirolimus; Sirolimus; Sirolimus (formerly known as rapamycin); Sirolimus (study drug)+ace inhibitor + statin; Sirolimus 0,1% crème; Sirolimus 0.2%; Sirolimus 0.4%; Sirolimus 1mg/ml; Sirolimus 2 mg; Sirolimus 2%; Sirolimus and hydroxychloroquine 200 mg; Sirolimus and hydroxychloroquine 400 mg; Sirolimus ointment; Sirolimus oral liquid product 1mg/ml; Sirolimus oral product; Sirolimus, 2%; Skincerity plus sirolimus/rapamycin; Skinercity plus sirolimus/rapamycin; Tavt-18 (sirolimus); Temsirolimus; Temsirolimus tablets; Topical 0.1% sirolimus;	[1] D00753 💬	[1] MTOR 💬	[50] AMPK signaling pathway, Acute myeloid leukemia, Adipocytokine signaling pathway, Alzheimer disease, Amyotrophic lateral sclerosis, Apelin signaling pathway, Autophagy - animal, Autophagy - other, Breast cancer, Cellular senescence, Central carbon metabolism in cancer, Choline metabolism in cancer, Colorectal cancer, Diabetic cardiomyopathy, EGFR tyrosine kinase inhibitor resistance, Endocrine resistance, ErbB signaling pathway, Gastric cancer, Glioma, Growth hormone synthesis, secretion and action, HIF-1 signaling pathway, Hepatocellular carcinoma, Herpes simplex virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Human papillomavirus infection, Huntington disease, Insulin resistance, Insulin signaling pathway, JAK-STAT signaling pathway, Kaposi sarcoma-associated herpesvirus infection, Longevity regulating pathway, Longevity regulating pathway - multiple species, MicroRNAs in cancer, Neutrophil extracellular trap formation, PD-L1 expression and PD-1 checkpoint pathway in cancer, PI3K-Akt signaling pathway, Pancreatic cancer, Pathways in cancer, Pathways of neurodegeneration - multiple diseases, Phospholipase D signaling pathway, Prostate cancer, Proteoglycans in cancer, Shigellosis, Spinocerebellar ataxia, Th17 cell differentiation, Thermogenesis, Thyroid hormone signaling pathway, Type II diabetes mellitus, mTOR signaling pathway	[40] 2 , 6 , 13, 15, 17, 19, 34, 35, 36, 46, 49, 51, 60, 61, 62, 63, 65, 66, 67, 84, 85, 86, 89, 96, 98, 127, 137, 157, 158, 192, 222, 256, 277, 278, 279, 280, 281, 283, 285, 331 💬
7	Water	[31] 15 o water; 15-o labeled water; 5% glucose water solution; Behavioral: diet and water adjustment; Behavioral: water tracking; Cholecalciferol concentrate (water-dispersible form); Device: manual toothbrush and water flosser (philips sonicare airfloss); Device: sonic toothbrush (philips sonicare) and water flosser (philips sonicare airfloss); Dextrose, 5% in water; High water intake; Lactose in water; Other: ad libitum water intake; Other: dead sea solar and water treatment; Other: dextrose water 5%; Other: exercise followed cold water immersion; Other: harkány medicinal water; Other: high water intake; Other: potable water; Other: water; Other: water prescription; Pl 12185/0002 & pl 12185/0005(water for injection); Procedure: water exercises; Pseudoephedrine + 480 ml water; Pseudoephedrine + 50 ml water; Sterile water; Sterile water for injection; Sterilised water for injections; Water; Water for injection; Water for injections; Water for injections, ep;	[1] D00001 💬	-	-	[22] 2 , 5 , 6 , 13, 17, 19, 35, 46, 51, 53, 56, 57, 63, 65, 67, 78, 84, 96, 97, 226, 278, 299 💬

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