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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: Nusinersen 💬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 Phosphate2件: Amifampridine,
Phosphate ion		1件: Amifampridine 💬4件: KCNA4,
KCNC3,
KCND2,
KCND3 💬		4件: Cortisol synthesis and secretion, Cushing syndrome, Serotonergic synapse, Spinocerebellar ataxia 💬1件: 3 💬
64-Aminopyridine1件: Dalfampridine1件: Dalfampridine 💬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: Amifampridine 💬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: Amifampridine 💬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: Amifampridine 💬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: Apitegromab 💬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: Branaplam 💬1件: SMN2 💬-2件: 3, 8 💬
20Celecoxib1件: Celecoxib1件: Celecoxib 💬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: Risdiplam 💬1件: SMN2 💬1件: Nucleocytoplasmic transport 💬1件: 3 💬
23EXG001-307 injection----1件: 3 💬
24Firdapse1件: Amifampridine1件: Amifampridine 💬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: Amifampridine 💬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: Hydroxyurea 💬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: Itraconazole 💬--6件: 3, 46, 65, 96, 227, 299 💬
34Leuprolide1件: Leuprolide---5件: 1, 2, 3, 51, 76 💬
35LEVOCARNITINE1件: Levocarnitine1件: Levocarnitine 💬--3件: 3, 53, 97 💬
36LMI070----2件: 3, 8 💬
37Mestinon1件: Pyridostigmine---3件: 3, 6, 11 💬
38Midazolam1件: Midazolam1件: Midazolam 💬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 SimpleXx1件: Somatotropin---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: Nusinersen 💬1件: SMN2 💬-1件: 3 💬
45Nusinersen Injectable Product1件: Nusinersen1件: Nusinersen 💬1件: SMN2 💬-1件: 3 💬
46NUSINERSEN SODIUM1件: Nusinersen1件: Nusinersen 💬1件: SMN2 💬-1件: 3 💬
47Nusinersen Treatments1件: Nusinersen1件: Nusinersen 💬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: Olesoxime 💬--3件: 2, 3, 13 💬
52ONASEMNOGENE ABEPARVOVEC1件: Onasemnogene abeparvovec1件: Onasemnogene abeparvovec 💬1件: SMN1 💬-1件: 3 💬
53Onasemnogene Abeparvovec-xioi1件: Onasemnogene abeparvovec1件: Onasemnogene abeparvovec 💬1件: SMN1 💬-1件: 3 💬
54Orfiril Saft----1件: 3 💬
55PARACETAMOL1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, 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: Acetaminophen 💬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: Prednisolone 💬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: Prednisolone 💬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: Reldesemtiv 💬1件: TNNC2 💬1件: Calcium signaling pathway 💬2件: 2, 3 💬
66Reldesemtiv 150 mg1件: Reldesemtiv1件: Reldesemtiv 💬1件: TNNC2 💬1件: Calcium signaling pathway 💬1件: 3 💬
67Reldesemtiv 450 mg1件: Reldesemtiv1件: Reldesemtiv 💬1件: TNNC2 💬1件: Calcium signaling pathway 💬1件: 3 💬
68RILUZOLE1件: Riluzole1件: Riluzole 💬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: Risdiplam 💬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: Albuterol 💬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: Sesame oil 💬--1件: 3 💬
88SESAME OIL, REFINED1件: Sesame oil1件: Sesame oil 💬--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: Nusinersen 💬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: Taldefgrobep alfa 💬1件: MSTN 💬1件: Cytokine-cytokine receptor interaction 💬1件: 3 💬
99Testosterone1件: Testosterone1件: Testosterone 💬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: Valproic acid 💬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: Valproic acid 💬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: Valproic acid ,
Levocarnitine 💬		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: Valproic acid 💬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: Valproic acid 💬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: Valproic acid ,
Levocarnitine 💬		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: Onasemnogene abeparvovec 💬1件: SMN1 💬-1件: 3 💬
110Zolgensma 2 x 1013 vector genomes/mL solution for infusion1件: Onasemnogene abeparvovec1件: Onasemnogene abeparvovec 💬1件: SMN1 💬-1件: 3 💬
111Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion1件: Onasemnogene abeparvovec1件: Onasemnogene abeparvovec 💬1件: SMN1 💬-1件: 3 💬