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	2,2-bis(4-hydroxy-3-tert-butylphenyl)propane	-	-	-	-	[4] 19, 86, 276, 299 💬
2	Abatacept	[36] Abatacept; Abatacept (aba); Abatacept (aba) + methotrexate (mtx), double-blind (db); Abatacept (bms-188667); Abatacept (ctla4-ig); Abatacept (iv); Abatacept (sc); Abatacept + csdmard; Abatacept + methotrexate; Abatacept + vaccines; Abatacept active treatment; Abatacept delayed-onset treatment; Abatacept injection; Abatacept placebo; Abatacept prefilled syringe; Abatacept sc; Abatacept subcutaneous; Abatacept, rituximab or tocilizumab; Adalimuab, etanercept, tocilizumab, or abatacept; Bdmard withdrawal (etanercept, adalimumab, infliximab, certolizumab, golimumab, tozilizumab, abatacept); Bms 188667 (abatacept); Decrease tocilizumab, abatacept; Device: abatacept combination product (acp); Double-blind abatacept; Drug: abatacept; Etanercepet, rituximab, abatacept, tocilizumab, sarilumab; Intravenous (iv) abatacept; Iv abatacept; Open-label abatacept; Orencia (abatacept); Orencia abatacept; Orencia® (abatacept); Subcutaneous (sc) abatacept; Subcutaneous abatacept; Subcutaneous(sc) abatacept; Tocilizumab, abatacept;	[1] D03203 💬	[2] CD80, CD86 💬	[12] Allograft rejection, Autoimmune thyroid disease, Cell adhesion molecules, Graft-versus-host disease, Intestinal immune network for IgA production, Kaposi sarcoma-associated herpesvirus infection, Rheumatoid arthritis, Systemic lupus erythematosus, Toll-like receptor signaling pathway, Transcriptional misregulation in cancer, Type I diabetes mellitus, Viral myocarditis	[26] 11, 13, 19, 41, 44, 46, 49, 50, 51, 53, 55, 60, 65, 84, 93, 95, 96, 97, 107, 160, 164, 222, 271, 284, 285, 300 💬
3	Acetylcysteine	[12] Acetylcysteine; Acetylcysteine effervescent tablets; Acetylcysteine sodium; Fluimucil (acetylcysteine); Glatiramer acetate, n-acetylcysteine; Jaktinib dihydrochloride monohydrate 50mg bid and mimic tablets of jakitinib hydrochloride 75mg bid and acetylcysteine effervescent tablets; Jaktinib dihydrochloride monohydrate 75mg bid and mimic tablets of jakitinib hydrochloride 50mg bid and acetylcysteine effervescent tablets; N acetylcysteine; N-acetylcysteine; N-acetylcysteine (nac); N-acetylcysteine capsule; N-acetylcysteine, (nac);	[1] D00221 💬	-	-	[17] 6 , 13, 19, 20, 34, 49, 51, 58, 85, 90, 94, 111, 164, 193, 233, 298, 299 💬
4	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 💬
5	Adrabetadex	[1] Adrabetadex;	-	-	-	[1] 19 💬
6	Afegostat	[2] Afegostat; Afegostat tartrate;	[1] D09576 💬	-	-	[1] 19 💬
7	Agalsidase alfa	[3] Agalsidase alfa; Replagal (agalsidase alfa); Replagal agalsidase alfa;	[1] D02784 💬	[1] GLA 💬	[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism	[1] 19 💬
8	Agalsidase beta	[4] Agalsidase beta; Agalsidase beta (gz419828); Agalsidase beta (recombinant form); Fabrazyme (agalsidase beta);	[1] D03228 💬	[1] GLA 💬	[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism	[1] 19 💬
9	Alemtuzumab	[20] Alemtuzumab; Alemtuzumab (already received by alemtuzumab cohort between 1991 and 1994; Alemtuzumab (already received by alemtuzumab cohort between 1991 and 1994); Alemtuzumab (campath ); Alemtuzumab (campath); Alemtuzumab (gz402673); Alemtuzumab 0.2 mg; Alemtuzumab 0.3 mg; Alemtuzumab 12 mg; Alemtuzumab 24 mg; Alemtuzumab and rituximab; Alemtuzumab gz402673; Alemtuzumab immunotherapy; Alemtuzumab injection; Alemtuzumab injection [lemtrada]; Alemtuzumab, rituximab; Campath-1h (alemtuzumab); Filgrastim, alemtuzumab; Transplant conditioning with mobilization and alemtuzumab; Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan;	[1] D02802 💬	-	-	[21] 13, 15, 19, 20, 43, 44, 46, 49, 51, 60, 62, 63, 65, 85, 96, 164, 234, 283, 284, 285, 326 💬
10	Alendronic acid	[1] Alendronic acid;	[2] D00939 D07119 💬	[1] FDPS 💬	[4] Human T-cell leukemia virus 1 infection, Influenza A, Metabolic pathways, Terpenoid backbone biosynthesis	[7] 19, 41, 46, 50, 271, 274, 299 💬

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