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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
1.Nusinersen1件: Nusinersen1件: D10881 1件: SMN2 💬-1件: 3
20000----1件: 3
32011-2012 seasonal flu vaccine Intramuscular----2件: 3, 113
42011-2012 seasonal flu vaccine Subcutaneous----2件: 3, 113
53,4-Diaminopyridine Phosphate1件: Phosphate ion---1件: 3
64-Aminopyridine1件: Dalfampridine1件: D04127 4件: KCNA4,
KCNC3,
KCND2,
KCND3 💬		4件: Cortisol synthesis and secretion, Cushing syndrome, Serotonergic synapse, Spinocerebellar ataxia 💬2件: 3, 13
74-cholesten-3-one, oxime----2件: 2, 3
84-cholesten-3-one,oxime----1件: 3
9Adipose derived mesenchymal stem cell----1件: 3
10AMIFAMPRIDINA----2件: 3, 11
11Amifampridina fosfato----2件: 3, 11
12Amifampridine1件: Amifampridine1件: D10228 4件: KCNA4,
KCNC3,
KCND2,
KCND3 💬		4件: Cortisol synthesis and secretion, Cushing syndrome, Serotonergic synapse, Spinocerebellar ataxia 💬3件: 3, 11, 12
13Amifampridine Phosphate2件: Amifampridine,
Phosphate ion		1件: D10228 4件: KCNA4,
KCNC3,
KCND2,
KCND3 💬		4件: Cortisol synthesis and secretion, Cushing syndrome, Serotonergic synapse, Spinocerebellar ataxia 💬3件: 3, 11, 12
14Amifampridine Phosphate 10 MG Oral Tablet2件: Amifampridine,
Phosphate ion		1件: D10228 4件: KCNA4,
KCNC3,
KCND2,
KCND3 💬		4件: Cortisol synthesis and secretion, Cushing syndrome, Serotonergic synapse, Spinocerebellar ataxia 💬1件: 3
15Apitegromab1件: Apitegromab1件: D11797 1件: MSTN 💬1件: Cytokine-cytokine receptor interaction 💬1件: 3
16AVXS-101----1件: 3
17AVXS-101 (previously known as scAAV9.CB.SMN)----1件: 3
18Biocarn----1件: 3
19Branaplam1件: Branaplam1件: D12272 1件: SMN2 💬-2件: 3, 8
20Celecoxib1件: Celecoxib1件: D00567 1件: PTGS2 💬22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, Regulation of lipolysis in adipocytes, Retrograde endocannabinoid signaling, Serotonergic synapse, Small cell lung cancer, TNF signaling pathway, VEGF signaling pathway 💬10件: 2, 3, 34, 46, 70, 89, 107, 161, 171, 271
21Disease modifying treatments----1件: 3
22EVRYSDI1件: Risdiplam1件: D11406 1件: SMN2 💬1件: Nucleocytoplasmic transport 💬1件: 3
23EXG001-307 injection----1件: 3
24Firdapse1件: Amifampridine1件: D10228 4件: KCNA4,
KCNC3,
KCND2,
KCND3 💬		4件: Cortisol synthesis and secretion, Cushing syndrome, Serotonergic synapse, Spinocerebellar ataxia 💬2件: 3, 11
25FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE1件: Amifampridine1件: D10228 4件: KCNA4,
KCNC3,
KCND2,
KCND3 💬		4件: Cortisol synthesis and secretion, Cushing syndrome, Serotonergic synapse, Spinocerebellar ataxia 💬2件: 3, 11
26GH----1件: 3
27GYM329----1件: 3
28HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY----1件: 3
29Hydroxyurea1件: Hydroxyurea1件: D00341 1件: RRM2 💬7件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism, p53 signaling pathway 💬8件: 3, 13, 19, 20, 65, 85, 86, 284
30ISIS 396443----1件: 3
31ISIS 396443 (BIIB058)----1件: 3
32ISIS 396443, BIIB058----1件: 3
33Itraconazole1件: Itraconazole1件: D00350 --6件: 3, 46, 65, 96, 227, 299
34Leuprolide1件: Leuprolide---5件: 1, 2, 3, 51, 76
35LEVOCARNITINE1件: Levocarnitine1件: D02176 --3件: 3, 53, 97
36LMI070----2件: 3, 8
37Mestinon1件: Pyridostigmine---3件: 3, 6, 11
38Midazolam1件: Midazolam1件: D00550 16件: GABRA1,
GABRA2,
GABRA3,
GABRA4,
GABRA5,
GABRA6,
GABRB1,
GABRB2,
GABRB3,
GABRD,
GABRE,
GABRG1,
GABRG2,
GABRG3,
GABRP,
GABRQ 💬		7件: GABAergic synapse, Morphine addiction, Neuroactive ligand-receptor interaction, Nicotine addiction, Retrograde endocannabinoid signaling, Serotonergic synapse, Taste transduction 💬12件: 2, 3, 6, 13, 21, 46, 70, 96, 97, 272, 298, 299
39N.a.----10件: 3, 6, 8, 13, 46, 96, 97, 240, 256, 271
40Nerve Growth Factor1件: Nerve Growth Factor---3件: 3, 53, 90
41Norditropin SimpleXx----5件: 3, 78, 107, 193, 195
42Norditropin SimpleXx 15 mg/1.5 ml1件: Somatotropin---1件: 3
43Nursinersen----1件: 3
44NUSINERSEN1件: Nusinersen1件: D10881 1件: SMN2 💬-1件: 3
45Nusinersen Injectable Product1件: Nusinersen1件: D10881 1件: SMN2 💬-1件: 3
46NUSINERSEN SODIUM1件: Nusinersen1件: D10881 1件: SMN2 💬-1件: 3
47Nusinersen Treatments1件: Nusinersen1件: D10881 1件: SMN2 💬-1件: 3
48OAV101----1件: 3
49Okrido----1件: 3
50Okrido 6mg/mL oral solution----1件: 3
51Olesoxime1件: Olesoxime1件: D11213 --3件: 2, 3, 13
52ONASEMNOGENE ABEPARVOVEC1件: Onasemnogene abeparvovec1件: D11559 1件: SMN1 💬-1件: 3
53Onasemnogene Abeparvovec-xioi1件: Onasemnogene abeparvovec1件: D11559 1件: SMN1 💬-1件: 3
54Orfiril Saft----1件: 3
55PARACETAMOL1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 💬6件: 3, 6, 13, 35, 46, 49
56Paracetamol 120Mg/5mL Oral Suspension1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 💬1件: 3
57PHENYLBUTYRATE1件: Phenylbutyric acid---12件: 2, 3, 6, 8, 15, 93, 94, 244, 251, 297, 299, 338
58Phosphate1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
59Placebo----28件: 3, 5, 6, 11, 13, 14, 37, 40, 46, 49, 50, 51, 53, 63, 66, 67, 79, 85, 86, 88, 96, 97, 140, 144, 149, 226, 271, 276
60PREDNISOLONE1件: Prednisolone1件: D00472 1件: NR3C1 💬1件: Neuroactive ligand-receptor interaction 💬42件: 3, 11, 13, 14, 25, 26, 35, 38, 39, 40, 41, 43, 44, 45, 46, 49, 50, 51, 56, 60, 61, 63, 64, 65, 66, 75, 81, 84, 85, 95, 96, 97, 107, 113, 145, 162, 222, 269, 271, 296, 299, 300
61PREDNISOLONE SODIUM PHOSPHATE2件: Phosphate ion,
Prednisolone		1件: D00472 1件: NR3C1 💬1件: Neuroactive ligand-receptor interaction 💬3件: 3, 13, 97
62Previously termed sc.AAV9.CB.SMN and AVXS-101----1件: 3
63Pyridostigmine1件: Pyridostigmine---4件: 3, 6, 11, 256
64Pyridostigmine Bromide1件: Pyridostigmine---3件: 3, 6, 256
65RELDESEMTIV1件: Reldesemtiv1件: D11363 1件: TNNC2 💬1件: Calcium signaling pathway 💬2件: 2, 3
66Reldesemtiv 150 mg1件: Reldesemtiv1件: D11363 1件: TNNC2 💬1件: Calcium signaling pathway 💬1件: 3
67Reldesemtiv 450 mg1件: Reldesemtiv1件: D11363 1件: TNNC2 💬1件: Calcium signaling pathway 💬1件: 3
68RILUZOLE1件: Riluzole1件: D00775 14件: GRIA1,
GRIA2,
GRIA3,
GRIA4,
GRIK1,
GRIK2,
GRIK3,
GRIK4,
GRIK5,
GRIN1,
GRIN2A,
GRIN2B,
GRIN2C,
GRIN2D 💬		23件: Alcoholism, Alzheimer disease, Amphetamine addiction, Amyotrophic lateral sclerosis, Calcium signaling pathway, Circadian entrainment, Cocaine addiction, Dopaminergic synapse, Glutamatergic synapse, Huntington disease, Long-term depression, Long-term potentiation, Neuroactive ligand-receptor interaction, Nicotine addiction, Pathways of neurodegeneration - multiple diseases, Prion disease, Rap1 signaling pathway, Ras signaling pathway, Retrograde endocannabinoid signaling, Spinocerebellar ataxia, Systemic lupus erythematosus, Transcriptional misregulation in cancer, cAMP signaling pathway 💬9件: 2, 3, 5, 8, 13, 17, 18, 203, 206
69Risdiplam1件: Risdiplam1件: D11406 1件: SMN2 💬1件: Nucleocytoplasmic transport 💬1件: 3
70RO6885247----1件: 3
71RO6885247/F02----1件: 3
72RO6885247/F03----1件: 3
73RO7034067----1件: 3
74RO7034067 / F12----1件: 3
75RO7034067 / F13----1件: 3
76RO7034067/F06 + solvente (RO----1件: 3
77RO7034067/F06 with solvent (RO7034067/F08)----1件: 3
78RO7034067/F07 + solvente (RO----1件: 3
79RO7034067/F07 with solvent (RO7034067/F09)----1件: 3
80RO7034067/F12----1件: 3
81RO7034067/F13----1件: 3
82RO7090919----1件: 3
83RO7204239----2件: 3, 113
84RO7204239/F01-01----1件: 3
85RVG 03820----1件: 3
86Salbutamol1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
87Sesame oil1件: Sesame oil1件: D03314 --1件: 3
88SESAME OIL, REFINED1件: Sesame oil1件: D03314 --1件: 3
89SMART----1件: 3
90Sodium phenylbutyrate1件: Phenylbutyric acid---4件: 2, 3, 8, 251
91Sodium phenylbutyrate (NaPB)1件: Phenylbutyric acid---1件: 3
92Somatotropin1件: Somatotropin---4件: 3, 65, 78, 236
93Somatropin1件: Somatotropin---19件: 2, 3, 19, 46, 65, 74, 75, 78, 96, 107, 113, 187, 193, 195, 238, 265, 274, 276, 299
94Spinraza1件: Nusinersen1件: D10881 1件: SMN2 💬-1件: 3
95SRK-015----1件: 3
96Sulfate1件: Sulfate ion---27件: 2, 3, 6, 11, 13, 26, 28, 34, 36, 46, 49, 53, 60, 64, 66, 85, 86, 96, 111, 218, 222, 224, 226, 265, 296, 297, 299
97Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide----1件: 3
98Taldefgrobep alfa1件: Talditercept alfa1件: D11265 1件: MSTN 💬1件: Cytokine-cytokine receptor interaction 💬1件: 3
99Testosterone1件: Testosterone1件: D00075 1件: AR 💬4件: Chemical carcinogenesis - receptor activation, Oocyte meiosis, Pathways in cancer, Prostate cancer 💬8件: 1, 2, 3, 13, 60, 76, 78, 113
100TRO19622----2件: 2, 3
101Valproate1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
102VALPROATE SODIUM1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 💬1件: 3
103Valproate, Levocarnitine2件: Levocarnitine,
Valproic acid		2件: D00399 ,
D02176 		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 💬1件: 3
104Valproic 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
105Valproic Acid (VPA)1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 💬1件: 3
106Valproic Acid and Levocarnitine2件: Levocarnitine,
Valproic acid		2件: D00399 ,
D02176 		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 💬1件: 3
107VOLMAX*30CPR 4MG R.P.----1件: 3
108VPA----1件: 3
109Zolgensma1件: Onasemnogene abeparvovec1件: D11559 1件: SMN1 💬-1件: 3
110Zolgensma 2 x 1013 vector genomes/mL solution for infusion1件: Onasemnogene abeparvovec1件: D11559 1件: SMN1 💬-1件: 3
111Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion1件: Onasemnogene abeparvovec1件: D11559 1件: SMN1 💬-1件: 3