Drug Description in Trials / DrugBank / KEGG DRUG

  

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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-deoxynojirimycin hydrochloride1件: Duvoglustat2件: Duvoglustat ,
Duvoglustat hydrochloride 💬 		1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
2[AT2221]1件: Miglustat1件: Miglustat 💬 1件: UGCG 💬 2件: Cornified envelope formation, Sphingolipid metabolism 💬1件: 256 💬
3[ATB200]----1件: 256 💬
4[GZ402666]----1件: 256 💬
5[Myozyme]----1件: 256 💬
6A-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA)----1件: 256 💬
7ABX11001件: ABX1100---1件: 256 💬
8ABX1100 injection for IV infusion1件: ABX1100---1件: 256 💬
9Acetaminophen1件: Acetaminophen1件: Acetaminophen 💬 2件: PTGS1,
PTGS2 💬 		23件: Alzheimer disease, Arachidonic acid metabolism, C-type lectin receptor signaling pathway, Chemical carcinogenesis - DNA adducts, Efferocytosis, Human cytomegalovirus infection, Human papillomavirus infection, IL-17 signaling pathway, Kaposi sarcoma-associated herpesvirus infection, Leishmaniasis, 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 💬19件: 11, 13, 19, 34, 35, 46, 49, 51, 63, 70, 77, 86, 96, 222, 231, 256, 271, 300, 337 💬
10ACTUS-1011件: ACTUS-101---1件: 256 💬
11Albuterol1件: Albuterol2件: Albuterol sulfate ,
Albuterol 💬 		1件: ADRB2 💬 11件: Adrenergic signaling in cardiomyocytes, Calcium signaling pathway, Chemical carcinogenesis - receptor activation, Hormone signaling, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction, Regulation of lipolysis in adipocytes, Renin secretion, Salivary secretion, cAMP signaling pathway, cGMP-PKG signaling pathway 💬9件: 12, 13, 86, 89, 113, 231, 256, 299, 340 💬
12Aldurazyme (laronidase)1件: Laronidase1件: Laronidase 💬 1件: IDUA 💬 2件: Glycosaminoglycan degradation, Lysosome biogenesis 💬2件: 19, 256 💬
13ALGLUCOSIDASE ALFA1件: Alglucosidase alfa1件: Alglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
14Alglucosidase alfa (GZ419829)1件: Alglucosidase alfa1件: Alglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
15ALGLUCOSIDASE ALFA (MYOZYME)1件: Alglucosidase alfa1件: Alglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
16Alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])1件: Alglucosidase alfa1件: Alglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
17Alglucosidase alfa GZ4198291件: Alglucosidase alfa1件: Alglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
18Alglucosidase alfa or Avalglucosidase alfa2件: Alglucosidase alfa,
Avalglucosidase alfa		2件: Alglucosidase alfa ,
Avalglucosidase alfa 💬 		1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
19Alglucosidase alpha----1件: 256 💬
20ALGLUCOSIDASI ALFA UMANA RICOMBINANTE----1件: 256 💬
21Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks----1件: 256 💬
22AT22201件: AT2220---1件: 256 💬
23AT22211件: Miglustat1件: Miglustat 💬 1件: UGCG 💬 2件: Cornified envelope formation, Sphingolipid metabolism 💬1件: 256 💬
24AT2221 65 mg Formulated Capsules1件: Miglustat1件: Miglustat 💬 1件: UGCG 💬 2件: Cornified envelope formation, Sphingolipid metabolism 💬1件: 256 💬
25AT8451件: AT845---1件: 256 💬
26ATB200----1件: 256 💬
27Avalglucosidase Alfa1件: Avalglucosidase alfa1件: Avalglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
28Avalglucosidase alfa (GZ402666)1件: Avalglucosidase alfa1件: Avalglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
29Avalglucosidase Alfa (NeoGAA)1件: Avalglucosidase alfa1件: Avalglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
30Avalglucosidase alfa-NGPT (GZ402666)1件: Avalglucosidase alfa1件: Avalglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
31Avalglucosidase alfa-NGPT (GZ402666) IV1件: Avalglucosidase alfa1件: Avalglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
32Avalglucosidasi alfa----1件: 256 💬
33Beta-hydroxybuturate esters----1件: 256 💬
34Blood test----19件: 2, 6, 13, 36, 46, 49, 51, 74, 79, 83, 96, 113, 127, 162, 227, 256, 280, 299, 300 💬
35BMN 701----1件: 256 💬
36BORTEZOMIB1件: Bortezomib1件: Bortezomib 💬 1件: PSMB5 💬 8件: Alzheimer disease, Amyotrophic lateral sclerosis, Huntington disease, Parkinson disease, Pathways of neurodegeneration - multiple diseases, Prion disease, Proteasome, Spinocerebellar ataxia 💬19件: 11, 13, 26, 28, 46, 49, 51, 61, 63, 64, 66, 172, 222, 223, 228, 256, 283, 288, 331 💬
37Calcium1件: Calcium---39件: 2, 3, 6, 13, 15, 19, 46, 48, 49, 51, 53, 56, 62, 67, 68, 71, 74, 75, 79, 84, 90, 93, 94, 95, 96, 97, 156, 186, 214, 215, 222, 224, 235, 256, 265, 271, 274, 298, 299 💬
38Cipaglucosidase Alfa1件: Cipaglucosidase alfa1件: Cipaglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
39Cipaglucosidasi alfa----1件: 256 💬
40Clenbuterol1件: Clenbuterol2件: Clenbuterol hydrochloride ,
Clenbuterol 💬 		1件: ADRB2 💬 11件: Adrenergic signaling in cardiomyocytes, Calcium signaling pathway, Chemical carcinogenesis - receptor activation, Hormone signaling, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction, Regulation of lipolysis in adipocytes, Renin secretion, Salivary secretion, cAMP signaling pathway, cGMP-PKG signaling pathway 💬6件: 1, 2, 6, 57, 113, 256 💬
41Creatine1件: Creatine---18件: 1, 2, 3, 5, 6, 8, 13, 15, 21, 46, 49, 50, 97, 113, 156, 201, 256, 337 💬
42Cyclophosphamide1件: Cyclophosphamide2件: Cyclophosphamide ,
Cyclophosphamide 💬 		--50件: 11, 13, 14, 16, 19, 20, 26, 28, 34, 35, 36, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 53, 56, 60, 61, 62, 63, 65, 66, 84, 93, 96, 162, 164, 172, 205, 221, 222, 223, 224, 256, 265, 274, 283, 284, 285, 288, 326, 331 💬
43CYCLOPHOSPHAMIDE MONOHYDRATE1件: Cyclophosphamide2件: Cyclophosphamide ,
Cyclophosphamide 💬 		--4件: 13, 60, 96, 256 💬
44Deprakine----1件: 256 💬
45Diagnostic Test: Beck depression inventory fast screen (Questionnaire)----3件: 3, 15, 256 💬
46Diagnostic Test: Borg Scale----3件: 3, 15, 256 💬
47Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire)----3件: 3, 15, 256 💬
48Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire)----3件: 3, 15, 256 💬
49Diagnostic Test: German Pain Inventory (Questionnaire)----3件: 3, 15, 256 💬
50Diagnostic Test: Handheld Dynamometry (HHD)----3件: 3, 15, 256 💬
51Diagnostic Test: Laboratory assessment: calcium1件: Calcium---3件: 3, 15, 256 💬
52Diagnostic Test: Laboratory assessment: Creatine kinase1件: Creatine---3件: 3, 15, 256 💬
53Diagnostic Test: Laboratory assessment: magnesium1件: Magnesium---3件: 3, 15, 256 💬
54Diagnostic Test: Laboratory assessment: phosphate----3件: 3, 15, 256 💬
55Diagnostic Test: Laboratory assessment: Vitamin D Level1件: Vitamin D---3件: 3, 15, 256 💬
56Diagnostic Test: Muscle ultrasound----5件: 3, 15, 111, 113, 256 💬
57Diagnostic Test: Neutralizing Antibody to SPK-3006 capsid----1件: 256 💬
58Diagnostic Test: Pressure pain threshold----4件: 3, 15, 113, 256 💬
59Diagnostic Test: Quick Motor Function Test----4件: 3, 15, 113, 256 💬
60Diagnostic Test: R-PAct (Questionnaire)----3件: 3, 15, 256 💬
61Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire)----3件: 3, 15, 256 💬
62Diagnostic Test: Six-minute walk test (6MWT)----3件: 3, 15, 256 💬
63Diagnostic Test: Vital signs----3件: 3, 15, 256 💬
64Diphenhydramine1件: Diphenhydramine4件: Diphenhydramine ,
Diphenhydramine hydrochloride ,
Diphenhydramine salicylate ,
Diphenhydramine citrate 💬 		1件: HRH1 💬 4件: Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction 💬12件: 11, 13, 19, 49, 51, 63, 70, 86, 96, 222, 256, 300 💬
65DNL952----1件: 256 💬
66Duvoglustat1件: Duvoglustat2件: Duvoglustat ,
Duvoglustat hydrochloride 💬 		1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
67Duvoglustat HCl1件: Duvoglustat2件: Duvoglustat ,
Duvoglustat hydrochloride 💬 		1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
68Duvoglustat hydrochloride1件: Duvoglustat2件: Duvoglustat ,
Duvoglustat hydrochloride 💬 		1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
69Enzyme Replacement Agent----2件: 19, 256 💬
70Filgrastim1件: Filgrastim1件: Filgrastim 💬 1件: CSF3R 💬 5件: Cytokine-cytokine receptor interaction, Hematopoietic cell lineage, JAK-STAT signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer 💬30件: 2, 6, 13, 25, 26, 28, 34, 35, 38, 40, 41, 42, 45, 46, 47, 49, 51, 60, 62, 63, 65, 96, 113, 164, 256, 283, 284, 285, 296, 331 💬
71Genetic: AB-1009 (GAA Gene)----1件: 256 💬
72Genetic: ACTUS-1011件: ACTUS-101---1件: 256 💬
73Genetic: Blood draw for optional genetic exome sequencing----3件: 3, 15, 256 💬
74Genetic: CRG003 injection----1件: 256 💬
75Genetic: GC301----1件: 256 💬
76Genetic: Genetic test: ACE polymorphism----3件: 3, 15, 256 💬
77Genetic: Genetic test: ACTN3 polymorphism----3件: 3, 15, 256 💬
78Genetic: Laboratory Tests----2件: 113, 256 💬
79Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase----1件: 256 💬
80Genetic: SPK-3006----1件: 256 💬
81Genetic: zocaglusagene nuzaparvovec----1件: 256 💬
82GILT-rhGAA1件: Reveglucosidase alfa---1件: 256 💬
83Glucosidase alfa----1件: 256 💬
84Glycosade----3件: 65, 256, 257 💬
85GZ402666----1件: 256 💬
86GZ419829----1件: 256 💬
87Intravenous immune globulin----1件: 256 💬
88Ketocal 4:1 liquid Nutricia (intervention)----1件: 256 💬
89LARONIDASE1件: Laronidase1件: Laronidase 💬 1件: IDUA 💬 2件: Glycosaminoglycan degradation, Lysosome biogenesis 💬2件: 19, 256 💬
90Lidocaine1件: Lidocaine3件: Lidocaine ,
Lidocaine hydrochloride ,
Lidocaine hydrochloride monohydrate 💬 		6件: SCN1A,
SCN2A,
SCN3A,
SCN5A,
SCN8A,
SCN9A 💬 		5件: Adrenergic signaling in cardiomyocytes, Cadherin signaling, Dopaminergic synapse, IgSF CAM signaling, Taste transduction 💬14件: 6, 13, 22, 34, 46, 51, 70, 86, 107, 168, 226, 231, 256, 298 💬
91LMX 4 Topical Cream----1件: 256 💬
92Low carbohydrate ketogenic diet----1件: 256 💬
93Lumizyme®----1件: 256 💬
94Magnesium1件: Magnesium---23件: 3, 6, 11, 13, 15, 46, 64, 70, 71, 93, 97, 113, 166, 225, 234, 235, 256, 284, 291, 296, 297, 298, 340 💬
95Mannose1件: Mannose1件: Demannose 💬 --2件: 13, 256 💬
96Methotrexate1件: Methotrexate2件: Methotrexate ,
Methotrexate sodium 💬 		2件: DHFR,
DHFR2 💬 		3件: Folate biosynthesis, Folate transport and metabolism, One carbon pool by folate 💬41件: 11, 13, 14, 19, 34, 35, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 53, 54, 55, 56, 60, 62, 65, 84, 93, 96, 97, 107, 162, 164, 172, 223, 240, 256, 270, 271, 284, 285, 286, 326, 331 💬
97METHOTREXATE SODIUM1件: Methotrexate2件: Methotrexate ,
Methotrexate sodium 💬 		2件: DHFR,
DHFR2 💬 		3件: Folate biosynthesis, Folate transport and metabolism, One carbon pool by folate 💬2件: 46, 256 💬
98MIGLUSTAT1件: Miglustat1件: Miglustat 💬 1件: UGCG 💬 2件: Cornified envelope formation, Sphingolipid metabolism 💬3件: 19, 256, 299 💬
99Multi-ingredient supplement (PDT-MIS)----1件: 256 💬
100MYOZYME----1件: 256 💬
101Myozyme 50 mg powder for concentrate for solution for infusion----1件: 256 💬
102MYOZYME®----1件: 256 💬
103Myozyme® (alglucosidase alfa)1件: Alglucosidase alfa1件: Alglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
104N.a.----11件: 3, 6, 8, 13, 46, 63, 96, 97, 240, 256, 271 💬
105NEO-GAA----1件: 256 💬
106NeoGAA1件: Avalglucosidase alfa1件: Avalglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
107NeoGAA or avalglucosidase alfa1件: Avalglucosidase alfa1件: Avalglucosidase alfa 💬 1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
108Nexviadyme----1件: 256 💬
109NON APPLICABILE----3件: 49, 51, 256 💬
110Other: Blood sample----37件: 2, 11, 13, 34, 35, 41, 44, 46, 48, 49, 50, 51, 53, 58, 61, 63, 65, 84, 86, 96, 97, 113, 160, 162, 177, 222, 223, 227, 234, 238, 240, 256, 279, 299, 335, 340, 348 💬
111Other: Drawing blood spots from Newborns----1件: 256 💬
112Other: RMST----1件: 256 💬
113Other: Saline----8件: 2, 6, 46, 97, 98, 256, 274, 301 💬
114Other: Untreated----1件: 256 💬
115Pyridostigmine1件: Pyridostigmine1件: Pyridostigmine bromide 💬 1件: ACHE 💬 3件: Cholinergic synapse, Glycerophospholipid metabolism, Neuroactive ligand signaling 💬5件: 3, 6, 11, 51, 256 💬
116Pyridostigmine Bromide1件: Pyridostigmine1件: Pyridostigmine bromide 💬 1件: ACHE 💬 3件: Cholinergic synapse, Glycerophospholipid metabolism, Neuroactive ligand signaling 💬5件: 3, 6, 11, 51, 256 💬
117RAAV1-CMV-GAA (study agent) Administration----1件: 256 💬
118Rapamycin1件: Sirolimus1件: Sirolimus 💬 1件: MTOR 💬 52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, 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, Integrated stress response (ISR) signaling pathway, Integrin signaling, JAK-STAT signaling pathway, Kaposi sarcoma-associated herpesvirus infection, Longevity regulating pathway, Longevity regulating pathway - multiple species, Lysosome biogenesis, 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 💬13件: 2, 15, 49, 51, 53, 60, 63, 67, 96, 158, 256, 278, 279 💬
119RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN----1件: 256 💬
120Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)2件: Avalglucosidase alfa,
Mannose		2件: Avalglucosidase alfa ,
Demannose 💬 		1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
121Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA)2件: Avalglucosidase alfa,
Mannose		2件: Avalglucosidase alfa ,
Demannose 💬 		1件: GAA 💬 3件: Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬1件: 256 💬
122Recombinant human acid alfa-glucosidase (rhGAA)----1件: 256 💬
123Recombinant human acid alpha-glucosidase (rhGAA)----1件: 256 💬
124RECOMBINANT HUMAN ALFA-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN----1件: 256 💬
125REN0011件: ReN001---1件: 256 💬
126Reveglucosidase alfa1件: Reveglucosidase alfa---1件: 256 💬
127Reveglucosidase alpha----1件: 256 💬
128RhGAA----1件: 256 💬
129RhGAA, acid alpha glucosidase----1件: 256 💬
130Rituxan----5件: 46, 60, 63, 256, 288 💬
131RITUXIMAB1件: Rituximab1件: Rituximab 💬 1件: MS4A1 💬 1件: Hematopoietic cell lineage 💬54件: 11, 13, 14, 19, 26, 34, 35, 36, 40, 41, 42, 43, 44, 45, 46, 48, 49, 50, 51, 52, 53, 55, 56, 60, 61, 62, 63, 64, 65, 66, 83, 84, 85, 86, 88, 93, 94, 96, 97, 162, 172, 222, 223, 229, 256, 271, 274, 283, 284, 285, 288, 296, 300, 331 💬
132S-606001----1件: 256 💬
133Salbutamol----9件: 2, 3, 11, 85, 86, 228, 256, 299, 340 💬
134Sodium Valproate----3件: 2, 102, 256 💬
135SPK-3006----1件: 256 💬
136Ss-hydroxybuturate esters----1件: 256 💬
137Triheptanoin1件: Triheptanoin1件: Triheptanoin 💬 --12件: 2, 8, 65, 149, 156, 248, 256, 257, 317, 318, 324, 344 💬
138UX007----5件: 8, 248, 256, 257, 344 💬
139VAL-1221----2件: 256, 309 💬
140Vitamin D1件: Vitamin D---26件: 3, 6, 13, 15, 19, 41, 43, 44, 46, 49, 50, 68, 74, 93, 95, 96, 97, 98, 179, 222, 228, 235, 256, 274, 298, 299 💬
141ZAVESCA----3件: 19, 256, 299 💬
142Zavesca® Prescription----1件: 256 💬