DDrare: Database of Drug Development for Rare Diseases

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No.	Drug (Description in trials)	DrugBank	KEGG DRUG	KEGG GENES	KEGG PATHWAY On map, Yellow: Drug target genes	Disease ID
1	Rapamycin, sirolimus	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	[2] 89, 158 💬
2	Sirolimus	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	[35] 6 , 13, 17, 19, 34, 35, 36, 46, 49, 51, 60, 61, 62, 63, 65, 66, 67, 84, 85, 89, 98, 127, 137, 157, 158, 192, 222, 277, 278, 279, 280, 281, 283, 285, 331 💬
3	Sirolimus (formerly known as rapamycin)	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	[1] 35 💬
4	Sirolimus (study drug)+ace inhibitor + statin	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	[1] 66 💬
5	Sirolimus 0,1% crème	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	[2] 278, 280 💬
6	Sirolimus 0.2%	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	[1] 158 💬
7	Sirolimus 0.4%	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	[1] 158 💬
8	Sirolimus 1mg/ml	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	[2] 278, 280 💬
9	Sirolimus 2 mg	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	[1] 17 💬
10	Sirolimus 2%	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	[2] 36, 192 💬

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