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	Acitretin	[1] Acitretin;	[1] D02754 💬	[5] RARA, RARB, RXRA, RXRB, RXRG 💬	[17] Acute myeloid leukemia, Adipocytokine signaling pathway, Bile secretion, Estrogen signaling pathway, Gastric cancer, Hepatitis C, Non-alcoholic fatty liver disease, Non-small cell lung cancer, PI3K-Akt signaling pathway, PPAR signaling pathway, Parathyroid hormone synthesis, secretion and action, Pathways in cancer, Small cell lung cancer, Th17 cell differentiation, Thyroid cancer, Thyroid hormone signaling pathway, Transcriptional misregulation in cancer	[1] 37 💬
2	Adalimumab	[76] Adalimumab; Adalimumab (40 mg); Adalimumab (ada); Adalimumab (d2e7); Adalimumab (humira); Adalimumab (humira®); Adalimumab (up to 9 months exposure); Adalimumab - gp2017; Adalimumab - us licensed humira; Adalimumab 40 mg; Adalimumab 40 mg eow or ew; Adalimumab 40 mg sc every other week; Adalimumab 40 mg subcutaneous (sc) every other week (eow); Adalimumab 40mg q2w; Adalimumab 80 mg; Adalimumab and infliximab; Adalimumab humira; Adalimumab injection; Adalimumab injection [humira]; Adalimumab pfs and pen; Adalimumab prn; Adalimumab with methotrexate; Adalimumab, current formulation; Adalimumab, etanercept, golimumab or infliximab; Adalimumab, new formulation; Adalimumab, plus prednisone; Adalimumab, solution for injection 40 mg/ (0.8 ml) pre-filled syringe; Adalimumab-eu; Adalimumab-pfizer; Amgevita (adalimumab biosimilar ); Anti-tnf therapy (etanercept or adalimumab); Anti-tnf:adalimumab (subcutaneus); Application of adalimumab; Azathioprine or adalimumab and infliximab; Bdmard withdrawal (etanercept, adalimumab, infliximab, certolizumab, golimumab, tozilizumab, abatacept); Biosimilar product to adalimumab; Ct-p17 sc ai (adalimumab); Db adalimumab 40 mg eow; Db adalimumab 40 mg ew; Device: adalimumab delivered in current autoinjector; Device: adalimumab delivered in current syringe; Device: adalimumab delivered in physiolis autoinjector; Device: adalimumab delivered in physiolis syringe; Diagnostic test: adalimumab serum trough concentration; Diagnostic test: adalimumab trough concentration; Double-blind adalimumab; Etanercept or adalimumab; Fitc-adalimumab; Gp2017 (adalimumab biosimilar); Humira (adalimumab original); Humira (adalimumab); Humira (adalimumab) 40 mg in 0.4 ml s.c. injection; Humira (adalimumab) 40 mg s.c. injection; Humira (adalimumab) 40mg in 0.4 ml s.c. injection; Humira (adalimumab) 40mg in 0.4ml s.c.injection; Humira 40mg solution for injection in pre-filled syringe (adalimumab); Humira ® (adalimumab) 40mg/0.8ml pre-filled syringe; Humira® (adalimumab); Infliximab and adalimumab; Infliximab or adalimumab or golimumab or vedolizumab or ustekinumab; Infliximab, adalimumab, certolizumab pegol; Infliximab, etanercept, adalimumab; Methotrexate,adalimumab; New formulation adalimumab; Ol adalimumab 40 mg; Open-label adalimumab; Open-labeladalimumabrescue; Other: adalimumab; Other: adalimumab treatment; Other: lengthening adalimumab dosing interval; Other: measurement of drug (adalimumab/infliximab); Sb5 (proposed biosimilar to adalimumab); Start adalimumab in monotherapy; Tnf blockers (infliximab, adalimumab, etanercept); Treatment with adalimumab; Tumor necrosis factor inhibitor (etanercept, infliximab, adalimumab, and golimumab);	[1] D02597 💬	[1] TNF 💬	[65] AGE-RAGE signaling pathway in diabetic complications, Adipocytokine signaling pathway, African trypanosomiasis, Allograft rejection, Alzheimer disease, Amoebiasis, Amyotrophic lateral sclerosis, Antifolate resistance, Antigen processing and presentation, Apoptosis, Asthma, C-type lectin receptor signaling pathway, Chagas disease, Coronavirus disease - COVID-19, Cytokine-cytokine receptor interaction, Dilated cardiomyopathy, Epstein-Barr virus infection, Fc epsilon RI signaling pathway, Fluid shear stress and atherosclerosis, Graft-versus-host disease, Hematopoietic cell lineage, Hepatitis B, Hepatitis C, Herpes simplex virus 1 infection, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Human papillomavirus infection, Hypertrophic cardiomyopathy, IL-17 signaling pathway, Inflammatory bowel disease, Influenza A, Insulin resistance, Legionellosis, Leishmaniasis, MAPK signaling pathway, Malaria, NF-kappa B signaling pathway, NOD-like receptor signaling pathway, Natural killer cell mediated cytotoxicity, Necroptosis, Non-alcoholic fatty liver disease, Osteoclast differentiation, Pathogenic Escherichia coli infection, Pathways of neurodegeneration - multiple diseases, Pertussis, Prion disease, Proteoglycans in cancer, RIG-I-like receptor signaling pathway, Rheumatoid arthritis, Salmonella infection, Shigellosis, Sphingolipid signaling pathway, Systemic lupus erythematosus, T cell receptor signaling pathway, TGF-beta signaling pathway, TNF signaling pathway, Toll-like receptor signaling pathway, Toxoplasmosis, Tuberculosis, Type I diabetes mellitus, Type II diabetes mellitus, Viral protein interaction with cytokine and cytokine receptor, Yersinia infection, mTOR signaling pathway	[16] 19, 37, 40, 41, 46, 56, 84, 96, 97, 151, 160, 164, 222, 226, 269, 271 💬
3	Alefacept	[1] Alefacept;	[1] D02800 💬	[1] CD58 💬	[2] Cell adhesion molecules, Epstein-Barr virus infection	[6] 37, 60, 65, 164, 284, 285 💬
4	Anakinra	[8] Anakinra; Anakinra (kineret®); Anakinra (r-methuil-1ra); Anakinra / kineret; Anakinra and peg stnf-r1; Kineret® (anakinra); Kineret® or anakinra; Response to anakinra associated with methotrexate;	[1] D02934 💬	[2] IL1R1, IL1R2 💬	[15] Amoebiasis, Cytokine-cytokine receptor interaction, Fluid shear stress and atherosclerosis, Hematopoietic cell lineage, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Inflammatory mediator regulation of TRP channels, MAPK signaling pathway, NF-kappa B signaling pathway, Osteoclast differentiation, Pathogenic Escherichia coli infection, Prostate cancer, Shigellosis, Th17 cell differentiation, Transcriptional misregulation in cancer	[19] 2 , 13, 15, 19, 28, 37, 41, 46, 49, 50, 56, 65, 84, 86, 97, 106, 107, 266, 299 💬
5	Brodalumab	[2] Brodalumab; Brodalumab 210mg sc;	[1] D10061 💬	[1] IL17RA 💬	[2] Cytokine-cytokine receptor interaction, IL-17 signaling pathway	[2] 37, 51 💬
6	Calcitriol	[6] Calcitriol; Calcitriol & calcium; Delayed release 6mp or calcitriol vs. purinethol; Other: empiric use of calcium carbonate and calcitriol; Other: pth based calcium carbonate and calcitriol repletion; Rapamycin-calcitriol combination;	[1] D00129 💬	[1] VDR 💬	[4] Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis	[8] 37, 46, 49, 66, 96, 158, 235, 326 💬
7	Certolizumab pegol	[18] 99mtc-s-hynic certolizumab pegol; Certolizumab pegol; Certolizumab pegol (cdp870); Certolizumab pegol (cdp870, czp); Certolizumab pegol (cdp870, tradename cimzia); Certolizumab pegol (cdp870, tradename cimzia)(prefilled syringes at the dose of 200mg); Certolizumab pegol (czp); Certolizumab pegol (czp) 200 mg; Certolizumab pegol (czp) 400 mg; Certolizumab pegol (rinn); Certolizumab pegol + methotrexate (mtx); Certolizumab pegol / cimzia; Certolizumab pegol /cimzia; Certolizumab pegol 200 mg/ml [cimzia]; Certolizumab pegol 400 mg; Cimzia certolizumab pegol 200 mg/ml solution for injection; Infliximab, adalimumab, certolizumab pegol; Pharmacokinetics of certolizumab pegol;	[1] D03441 💬	[1] TNF 💬	[65] AGE-RAGE signaling pathway in diabetic complications, Adipocytokine signaling pathway, African trypanosomiasis, Allograft rejection, Alzheimer disease, Amoebiasis, Amyotrophic lateral sclerosis, Antifolate resistance, Antigen processing and presentation, Apoptosis, Asthma, C-type lectin receptor signaling pathway, Chagas disease, Coronavirus disease - COVID-19, Cytokine-cytokine receptor interaction, Dilated cardiomyopathy, Epstein-Barr virus infection, Fc epsilon RI signaling pathway, Fluid shear stress and atherosclerosis, Graft-versus-host disease, Hematopoietic cell lineage, Hepatitis B, Hepatitis C, Herpes simplex virus 1 infection, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Human papillomavirus infection, Hypertrophic cardiomyopathy, IL-17 signaling pathway, Inflammatory bowel disease, Influenza A, Insulin resistance, Legionellosis, Leishmaniasis, MAPK signaling pathway, Malaria, NF-kappa B signaling pathway, NOD-like receptor signaling pathway, Natural killer cell mediated cytotoxicity, Necroptosis, Non-alcoholic fatty liver disease, Osteoclast differentiation, Pathogenic Escherichia coli infection, Pathways of neurodegeneration - multiple diseases, Pertussis, Prion disease, Proteoglycans in cancer, RIG-I-like receptor signaling pathway, Rheumatoid arthritis, Salmonella infection, Shigellosis, Sphingolipid signaling pathway, Systemic lupus erythematosus, T cell receptor signaling pathway, TGF-beta signaling pathway, TNF signaling pathway, Toll-like receptor signaling pathway, Toxoplasmosis, Tuberculosis, Type I diabetes mellitus, Type II diabetes mellitus, Viral protein interaction with cytokine and cytokine receptor, Yersinia infection, mTOR signaling pathway	[6] 37, 46, 96, 97, 226, 271 💬
8	Efalizumab	[1] Efalizumab;	[1] D03959 💬	[1] ITGAL 💬	[12] Cell adhesion molecules, Epstein-Barr virus infection, Human T-cell leukemia virus 1 infection, Leukocyte transendothelial migration, Malaria, Natural killer cell mediated cytotoxicity, Neutrophil extracellular trap formation, Rap1 signaling pathway, Regulation of actin cytoskeleton, Rheumatoid arthritis, Staphylococcus aureus infection, Viral myocarditis	[9] 11, 13, 37, 46, 49, 53, 61, 95, 96 💬
9	Guselkumab	[7] Guselkumab; Guselkumab 100 mg; Guselkumab dose 1; Guselkumab dose 2; Guselkumab dose 3; Guselkumab dose 4; Guselkumab dose 5;	[1] D10438 💬	[1] IL23A 💬	[9] C-type lectin receptor signaling pathway, Cytokine-cytokine receptor interaction, Inflammatory bowel disease, JAK-STAT signaling pathway, Pathways in cancer, Pertussis, Rheumatoid arthritis, Th17 cell differentiation, Tuberculosis	[4] 37, 41, 96, 97 💬
10	Infliximab	[70] 99m technetium infliximab; 99mtc-infliximab; Abp 710 - biosimilar to infliximab; Adalimumab and infliximab; Adalimumab, etanercept, golimumab or infliximab; Anti tnf therapy including infliximab; Anti-tnf: infliximab (infusion); Azathioprine or adalimumab and infliximab; Bdmard withdrawal (etanercept, adalimumab, infliximab, certolizumab, golimumab, tozilizumab, abatacept); Biosimilar infliximab; Ct-p13 sc (infliximab); Cyclosporine vs infliximab; Inflectra (infliximab); Infliximab; Infliximab (bcd-055); Infliximab (ifx alone); Infliximab (ifx); Infliximab (inf) + mtx, db; Infliximab (remicade); Infliximab (remicade): 100 mg of lyophilized infliximab; Infliximab + methotrexate (mtx); Infliximab 10 mg/kg; Infliximab 20 mg/kg; Infliximab 3 mg/kg; Infliximab 5 mg/kg; Infliximab 5 mg/kg body weight infused over 2 hours; Infliximab 5 mg/kg every 6 weeks; Infliximab 7 mg/kg every 8 weeks; Infliximab [infliximab biosimilar 3]; Infliximab and adalimumab; Infliximab and azathioprine combination at week 0; Infliximab and azathioprine combination at week 14; Infliximab and mtx; Infliximab biosimilar; Infliximab control; Infliximab group; Infliximab increased dose; Infliximab increased frequency; Infliximab infusion; Infliximab or adalimumab or golimumab or vedolizumab or ustekinumab; Infliximab with azathioprine (iifx + aza); Infliximab+azathioprine; Infliximab, adalimumab, certolizumab pegol; Infliximab, azathioprine; Infliximab, etanercept; Infliximab, etanercept, adalimumab; Infliximab, methylprednisolone, methotrexate; Infliximab-biosimilar; Infliximab-eu; Infliximab-innovator; Infliximab-pfizer; Innovator infliximab; Intravitreal infliximab; Methotrexate + infliximab; Other: infliximab (ifx) infusion; Other: measurement of drug (adalimumab/infliximab); Procedure: measurement of serum infliximab and anti-infliximab antibodies; Procedure: treatment according to current standards without knowledge of serum infliximab and anti-infliximab ab status; Remicade (infliximab); Remicade® (infliximab); Remicaide (infliximab); Remsima (infliximab); Response to infliximab associated with methotrexate; Sb2 (infliximab biosimilar); Sb2 (proposed biosimilar to infliximab); Ta-650 (infliximab); Tnf blockers (infliximab, adalimumab, etanercept); Topical infliximab; Trexan+salazopyrin+oxiklorin+prednisolone + infliximab; Tumor necrosis factor inhibitor (etanercept, infliximab, adalimumab, and golimumab);	[1] D02598 💬	[1] TNF 💬	[65] AGE-RAGE signaling pathway in diabetic complications, Adipocytokine signaling pathway, African trypanosomiasis, Allograft rejection, Alzheimer disease, Amoebiasis, Amyotrophic lateral sclerosis, Antifolate resistance, Antigen processing and presentation, Apoptosis, Asthma, C-type lectin receptor signaling pathway, Chagas disease, Coronavirus disease - COVID-19, Cytokine-cytokine receptor interaction, Dilated cardiomyopathy, Epstein-Barr virus infection, Fc epsilon RI signaling pathway, Fluid shear stress and atherosclerosis, Graft-versus-host disease, Hematopoietic cell lineage, Hepatitis B, Hepatitis C, Herpes simplex virus 1 infection, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Human papillomavirus infection, Hypertrophic cardiomyopathy, IL-17 signaling pathway, Inflammatory bowel disease, Influenza A, Insulin resistance, Legionellosis, Leishmaniasis, MAPK signaling pathway, Malaria, NF-kappa B signaling pathway, NOD-like receptor signaling pathway, Natural killer cell mediated cytotoxicity, Necroptosis, Non-alcoholic fatty liver disease, Osteoclast differentiation, Pathogenic Escherichia coli infection, Pathways of neurodegeneration - multiple diseases, Pertussis, Prion disease, Proteoglycans in cancer, RIG-I-like receptor signaling pathway, Rheumatoid arthritis, Salmonella infection, Shigellosis, Sphingolipid signaling pathway, Systemic lupus erythematosus, T cell receptor signaling pathway, TGF-beta signaling pathway, TNF signaling pathway, Toll-like receptor signaling pathway, Toxoplasmosis, Tuberculosis, Type I diabetes mellitus, Type II diabetes mellitus, Viral protein interaction with cytokine and cytokine receptor, Yersinia infection, mTOR signaling pathway	[25] 35, 37, 38, 39, 40, 41, 42, 43, 45, 46, 49, 50, 55, 56, 65, 84, 85, 95, 96, 97, 98, 162, 164, 269, 271 💬

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