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No. 薬物名（臨床試験情報から抽出） DrugBank KEGG DRUG KEGG GENES KEGG PATHWAY 指定難病告示番号
11-deoxynojirimycin1件: Duvoglustat1件: D09605 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
21-deoxynojirimycin hydrochloride1件: Duvoglustat1件: D09605 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
33TC----2件: 256, 265
4A-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA)1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
5ABC----2件: 256, 265
6ABC/DTG/3TC STR - Tablet----1件: 256
7Acetaminophen1件: Acetaminophen1件: D00217 2件: PTGS1,
PTGS2 💬		23件: Alzheimer disease, Arachidonic acid metabolism, C-type lectin receptor signaling pathway, Chemical carcinogenesis - DNA adducts, Human cytomegalovirus infection, Human papillomavirus infection, IL-17 signaling pathway, Kaposi sarcoma-associated herpesvirus infection, Leishmaniasis, Metabolic pathways, MicroRNAs in cancer, NF-kappa B signaling pathway, Ovarian steroidogenesis, Oxytocin signaling pathway, Pathways in cancer, Pathways of neurodegeneration - multiple diseases, Platelet activation, Regulation of lipolysis in adipocytes, Retrograde endocannabinoid signaling, Serotonergic synapse, Small cell lung cancer, TNF signaling pathway, VEGF signaling pathway 💬13件: 11, 13, 34, 35, 46, 49, 51, 70, 86, 222, 256, 271, 337
8ACTUS-101----1件: 256
9Albuterol1件: Salbutamol1件: D02147 1件: ADRB2 💬9件: Adrenergic signaling in cardiomyocytes, Calcium signaling pathway, Chemical carcinogenesis - receptor activation, Neuroactive ligand-receptor interaction, Regulation of lipolysis in adipocytes, Renin secretion, Salivary secretion, cAMP signaling pathway, cGMP-PKG signaling pathway 💬7件: 12, 13, 86, 89, 113, 256, 299
10Aldurazyme (laronidase)1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬2件: 19, 256
11ALGLUCOSIDASE ALFA1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
12Alglucosidase Alfa (Genetical Recombination)1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
13Alglucosidase alfa (GZ419829)1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
14ALGLUCOSIDASE ALFA (MYOZYME)1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
15Alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
16Alglucosidase alfa GZ4198291件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
17Alglucosidase alpha----1件: 256
18ALGLUCOSIDASI ALFA UMANA RICOMBINANTE----1件: 256
19Alpha-glucosidase1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
20AT22201件: AT2220---1件: 256
21AT2221----1件: 256
22AT2221 65 mg Formulated Capsules----1件: 256
23AT845----1件: 256
24ATB200----1件: 256
25Avalglucosidase Alfa1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
26Avalglucosidase alfa (GZ402666)1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
27Avalglucosidase Alfa (NeoGAA)1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
28Avalglucosidase alfa GZ4026661件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
29Avalglucosidase alfa-NGPT (GZ402666)1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
30Avalglucosidase alfa-NGPT (GZ402666) IV1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
31Avalglucosidasi alfa----1件: 256
32Beta-hydroxybuturate esters----1件: 256
33BMN 701----1件: 256
34BORTEZOMIB1件: Bortezomib1件: D03150 1件: PSMB5 💬8件: Alzheimer disease, Amyotrophic lateral sclerosis, Huntington disease, Parkinson disease, Pathways of neurodegeneration - multiple diseases, Prion disease, Proteasome, Spinocerebellar ataxia 💬17件: 11, 13, 26, 28, 46, 49, 51, 61, 63, 64, 66, 222, 228, 256, 283, 288, 331
35Cabotegravir1件: Cabotegravir1件: D10548 --1件: 256
36Cabotegravir (CAB) tablet1件: Cabotegravir1件: D10548 --1件: 256
37Cabotegravir - Injectable Suspension (CAB LA)1件: Cabotegravir1件: D10548 --1件: 256
38Cipaglucosidase Alfa1件: Cipaglucosidase alfa1件: D11798 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
39Cipaglucosidasi alfa----1件: 256
40Clenbuterol1件: Clenbuterol1件: D07713 1件: ADRB2 💬9件: Adrenergic signaling in cardiomyocytes, Calcium signaling pathway, Chemical carcinogenesis - receptor activation, Neuroactive ligand-receptor interaction, Regulation of lipolysis in adipocytes, Renin secretion, Salivary secretion, cAMP signaling pathway, cGMP-PKG signaling pathway 💬4件: 1, 2, 6, 256
41Cyclophosphamide1件: Cyclophosphamide2件: D00287 ,
D07760 		--44件: 11, 13, 14, 16, 19, 20, 26, 28, 35, 36, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 56, 60, 62, 65, 66, 84, 85, 93, 96, 162, 164, 172, 205, 222, 224, 256, 274, 283, 284, 285, 288, 326, 331
42CYCLOPHOSPHAMIDE MONOHYDRATE1件: Cyclophosphamide2件: D00287 ,
D07760 		--4件: 13, 60, 96, 256
43Deoxynojirimycin1件: Duvoglustat1件: D09605 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
44Deprakine----1件: 256
45Diphenhydramine1件: Diphenhydramine1件: D00300 1件: HRH1 💬3件: Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand-receptor interaction 💬8件: 11, 13, 49, 51, 70, 86, 222, 256
46DTG Tablet----1件: 256
47Duvoglustat1件: Duvoglustat1件: D09605 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
48Duvoglustat HCl1件: Duvoglustat1件: D09605 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
49Duvoglustat hydrochloride1件: Duvoglustat1件: D09605 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
50Enzyme Replacement Agent----1件: 256
51Epilim----1件: 256
52Epilim Chrono 200 Controlled Released tablets----1件: 256
53Epilim Chrono 300 Controlled Released tablets----1件: 256
54Epilim Chrono 500 Controlled Released tablets----1件: 256
55Filgrastim1件: Filgrastim1件: D03235 1件: CSF3R 💬5件: Cytokine-cytokine receptor interaction, Hematopoietic cell lineage, JAK-STAT signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer 💬28件: 2, 6, 13, 25, 26, 28, 34, 35, 38, 40, 41, 42, 45, 46, 49, 51, 60, 62, 65, 96, 113, 164, 256, 283, 284, 285, 296, 331
56GC301----1件: 256
57GILT-rhGAA----1件: 256
58Glucosidase alfa----1件: 256
59Glycosade----3件: 65, 256, 257
60GZ402666----1件: 256
61GZ419829----1件: 256
62Intravenous immune globulin----1件: 256
63Ketocal 4:1 liquid Nutricia (intervention)----1件: 256
64LA----5件: 6, 13, 76, 78, 256
65Laboratory Tests----2件: 113, 256
66LARONIDASE1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬2件: 19, 256
67Lidocaine1件: Lidocaine2件: D00358 ,
D08127 		5件: SCN1A,
SCN2A,
SCN3A,
SCN5A,
SCN9A 💬		3件: Adrenergic signaling in cardiomyocytes, Dopaminergic synapse, Taste transduction 💬12件: 6, 13, 22, 34, 46, 51, 70, 86, 107, 168, 226, 256
68LMX 4 Topical Cream----1件: 256
69Low carbohydrate ketogenic diet----1件: 256
70Lumizyme1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
71Lumizyme®1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
72Mannose1件: Mannose---2件: 13, 256
73Methotrexate1件: Methotrexate1件: D00142 2件: DHFR,
DHFR2 💬		5件: Antifolate resistance, Biosynthesis of cofactors, Folate biosynthesis, Metabolic pathways, One carbon pool by folate 💬38件: 11, 13, 19, 34, 35, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 53, 55, 56, 60, 62, 65, 84, 93, 96, 97, 107, 162, 164, 172, 256, 270, 271, 283, 284, 285, 286, 326, 331
74METHOTREXATE SODIUM1件: Methotrexate1件: D00142 2件: DHFR,
DHFR2 💬		5件: Antifolate resistance, Biosynthesis of cofactors, Folate biosynthesis, Metabolic pathways, One carbon pool by folate 💬2件: 46, 256
75MIGLUSTAT1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 19, 256, 299
76Miglustat (AT2221)1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 256
77Myozyme1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
78Myozyme 50 mg powder for concentrate for solution for infusion1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
79MYOZYME®1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
80Myozyme® (alglucosidase alfa)1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
81N.a.----10件: 3, 6, 8, 13, 46, 96, 97, 240, 256, 271
82NEO-GAA----1件: 256
83NeoGAA1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
84NeoGAA or avalglucosidase alfa1件: Avalglucosidase alfa1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
85Non applicabile----4件: 13, 49, 51, 256
86Phosphate1件: Phosphate ion---32件: 2, 3, 6, 11, 12, 13, 28, 36, 46, 49, 51, 53, 60, 62, 65, 70, 78, 81, 83, 90, 96, 97, 164, 235, 238, 256, 276, 283, 284, 285, 286, 299
87Plecebo----1件: 256
88Pyridostigmine1件: Pyridostigmine---4件: 3, 6, 11, 256
89Pyridostigmine Bromide1件: Pyridostigmine---3件: 3, 6, 256
90RAAV1-CMV-GAA (study agent) Administration----1件: 256
91Rapamycin1件: Sirolimus1件: D00753 1件: MTOR 💬51件: 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, Chemical carcinogenesis - receptor activation, 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 💬18件: 15, 34, 35, 46, 49, 51, 53, 60, 61, 67, 86, 89, 96, 157, 158, 193, 256, 279
92Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
93RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN1件: Phosphate ion---1件: 256
94Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)3件: Avalglucosidase alfa,
Mannose,
Phosphate ion		1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
95Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA)3件: Avalglucosidase alfa,
Mannose,
Phosphate ion		1件: D11744 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
96Recombinant human acid alfa-glucosidase----1件: 256
97Recombinant human acid alfa-glucosidase (ATB200)----1件: 256
98Recombinant human acid alfa-glucosidase (rhGAA)----1件: 256
99Recombinant human acid alpha-glucosidase (rhGAA)1件: Alglucosidase alfa1件: D03207 1件: GAA 💬4件: Galactose metabolism, Lysosome, Metabolic pathways, Starch and sucrose metabolism 💬1件: 256
100RECOMBINANT HUMAN ALFA-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN1件: Phosphate ion---1件: 256
101REN0011件: ReN001---1件: 256
102Reveglucosidase alfa----1件: 256
103Reveglucosidase alpha----1件: 256
104RhGAA----1件: 256
105RhGAA, acid alpha glucosidase----1件: 256
106Rilpivirine1件: Rilpivirine1件: D09720 --1件: 256
107Rilpivirine (RPV) tablet1件: Rilpivirine1件: D09720 --1件: 256
108Rilpivirine - Injectable Suspension (RPV LA)1件: Rilpivirine1件: D09720 --1件: 256
109Rituxan----4件: 46, 60, 256, 288
110RITUXIMAB1件: Rituximab1件: D02994 1件: MS4A1 💬1件: Hematopoietic cell lineage 💬48件: 11, 13, 14, 19, 26, 35, 36, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 53, 55, 56, 60, 61, 63, 64, 65, 66, 83, 84, 85, 86, 93, 94, 96, 97, 162, 222, 229, 256, 271, 274, 283, 284, 285, 288, 296, 300, 331
111Salbutamol1件: Salbutamol1件: D02147 1件: ADRB2 💬9件: Adrenergic signaling in cardiomyocytes, Calcium signaling pathway, Chemical carcinogenesis - receptor activation, Neuroactive ligand-receptor interaction, Regulation of lipolysis in adipocytes, Renin secretion, Salivary secretion, cAMP signaling pathway, cGMP-PKG signaling pathway 💬8件: 3, 11, 49, 85, 86, 111, 256, 299
112Sodium Valproate1件: Valproic acid1件: D00399 7件: ABAT,
ALDH5A1,
CACNA1G,
CACNA1H,
CACNA1I,
GAD1,
GAD2 💬		17件: Alanine, aspartate and glutamate metabolism, Aldosterone synthesis and secretion, Butanoate metabolism, Calcium signaling pathway, Circadian entrainment, Cortisol synthesis and secretion, Cushing syndrome, GABAergic synapse, GnRH secretion, MAPK signaling pathway, Metabolic pathways, Propanoate metabolism, Taurine and hypotaurine metabolism, Type I diabetes mellitus, Type II diabetes mellitus, Valine, leucine and isoleucine degradation, beta-Alanine metabolism 💬4件: 2, 90, 102, 256
113SPK-3006----1件: 256
114Ss-hydroxybuturate esters----1件: 256
115Triheptanoin1件: Triheptanoin1件: D11465 --10件: 2, 8, 65, 149, 156, 248, 256, 257, 317, 324
116UX007----4件: 8, 248, 256, 257
117VAL-1221----1件: 256
118Valproate1件: Valproic acid1件: D00399 7件: ABAT,
ALDH5A1,
CACNA1G,
CACNA1H,
CACNA1I,
GAD1,
GAD2 💬		17件: Alanine, aspartate and glutamate metabolism, Aldosterone synthesis and secretion, Butanoate metabolism, Calcium signaling pathway, Circadian entrainment, Cortisol synthesis and secretion, Cushing syndrome, GABAergic synapse, GnRH secretion, MAPK signaling pathway, Metabolic pathways, Propanoate metabolism, Taurine and hypotaurine metabolism, Type I diabetes mellitus, Type II diabetes mellitus, Valine, leucine and isoleucine degradation, beta-Alanine metabolism 💬6件: 2, 3, 90, 102, 233, 256
119Valproic Acid1件: Valproic acid1件: D00399 7件: ABAT,
ALDH5A1,
CACNA1G,
CACNA1H,
CACNA1I,
GAD1,
GAD2 💬		17件: Alanine, aspartate and glutamate metabolism, Aldosterone synthesis and secretion, Butanoate metabolism, Calcium signaling pathway, Circadian entrainment, Cortisol synthesis and secretion, Cushing syndrome, GABAergic synapse, GnRH secretion, MAPK signaling pathway, Metabolic pathways, Propanoate metabolism, Taurine and hypotaurine metabolism, Type I diabetes mellitus, Type II diabetes mellitus, Valine, leucine and isoleucine degradation, beta-Alanine metabolism 💬7件: 3, 5, 26, 65, 90, 222, 256
120ZAVESCA1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 19, 256, 299
121Zavesca® Prescription1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 256