Drug Description in Trials / DrugBank / KEGG DRUG

  

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No. Drug (Description in trials) DrugBank KEGG DRUG KEGG GENES KEGG PATHWAY Disease ID
11-deoxynojirimycin hydrochloride[1] Duvoglustat[2] Duvoglustat ,
Duvoglustat hydrochloride 💬 		[1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
2[AT2221][1] Miglustat[1] Miglustat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 256 💬
3[ATB200]----[1] 256 💬
4[GZ402666]----[1] 256 💬
5[Myozyme]----[1] 256 💬
6A-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA)----[1] 256 💬
7ABX1100[1] ABX1100---[1] 256 💬
8ABX1100 injection for IV infusion[1] ABX1100---[1] 256 💬
9Acetaminophen[1] Acetaminophen[1] Acetaminophen 💬 [2] PTGS1,
PTGS2 💬 		[23] 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] 11, 13, 19, 34, 35, 46, 49, 51, 63, 70, 77, 86, 96, 222, 231, 256, 271, 300, 337 💬
10ACTUS-101[1] ACTUS-101---[1] 256 💬
11Albuterol[1] Albuterol[2] Albuterol sulfate ,
Albuterol 💬 		[1] ADRB2 💬 [11] 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] 12, 13, 86, 89, 113, 231, 256, 299, 340 💬
12Aldurazyme (laronidase)[1] Laronidase[1] Laronidase 💬 [1] IDUA 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[2] 19, 256 💬
13ALGLUCOSIDASE ALFA[1] Alglucosidase alfa[1] Alglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
14Alglucosidase alfa (GZ419829)[1] Alglucosidase alfa[1] Alglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
15ALGLUCOSIDASE ALFA (MYOZYME)[1] Alglucosidase alfa[1] Alglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
16Alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])[1] Alglucosidase alfa[1] Alglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
17Alglucosidase alfa GZ419829[1] Alglucosidase alfa[1] Alglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
18Alglucosidase alfa or Avalglucosidase alfa[2] Alglucosidase alfa,
Avalglucosidase alfa		[2] Alglucosidase alfa ,
Avalglucosidase alfa 💬 		[1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
19Alglucosidase alpha----[1] 256 💬
20ALGLUCOSIDASI ALFA UMANA RICOMBINANTE----[1] 256 💬
21Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks----[1] 256 💬
22AT2220[1] AT2220---[1] 256 💬
23AT2221[1] Miglustat[1] Miglustat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 256 💬
24AT2221 65 mg Formulated Capsules[1] Miglustat[1] Miglustat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 256 💬
25AT845[1] AT845---[1] 256 💬
26ATB200----[1] 256 💬
27Avalglucosidase Alfa[1] Avalglucosidase alfa[1] Avalglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
28Avalglucosidase alfa (GZ402666)[1] Avalglucosidase alfa[1] Avalglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
29Avalglucosidase Alfa (NeoGAA)[1] Avalglucosidase alfa[1] Avalglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
30Avalglucosidase alfa-NGPT (GZ402666)[1] Avalglucosidase alfa[1] Avalglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
31Avalglucosidase alfa-NGPT (GZ402666) IV[1] Avalglucosidase alfa[1] Avalglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
32Avalglucosidasi alfa----[1] 256 💬
33Beta-hydroxybuturate esters----[1] 256 💬
34Blood test----[19] 2, 6, 13, 36, 46, 49, 51, 74, 79, 83, 96, 113, 127, 162, 227, 256, 280, 299, 300 💬
35BMN 701----[1] 256 💬
36BORTEZOMIB[1] Bortezomib[1] Bortezomib 💬 [1] PSMB5 💬 [8] Alzheimer disease, Amyotrophic lateral sclerosis, Huntington disease, Parkinson disease, Pathways of neurodegeneration - multiple diseases, Prion disease, Proteasome, Spinocerebellar ataxia 💬[19] 11, 13, 26, 28, 46, 49, 51, 61, 63, 64, 66, 172, 222, 223, 228, 256, 283, 288, 331 💬
37Calcium[1] Calcium---[39] 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 Alfa[1] Cipaglucosidase alfa[1] Cipaglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
39Cipaglucosidasi alfa----[1] 256 💬
40Clenbuterol[1] Clenbuterol[2] Clenbuterol hydrochloride ,
Clenbuterol 💬 		[1] ADRB2 💬 [11] 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] 1, 2, 6, 57, 113, 256 💬
41Creatine[1] Creatine---[18] 1, 2, 3, 5, 6, 8, 13, 15, 21, 46, 49, 50, 97, 113, 156, 201, 256, 337 💬
42Cyclophosphamide[1] Cyclophosphamide[2] Cyclophosphamide ,
Cyclophosphamide 💬 		--[50] 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 MONOHYDRATE[1] Cyclophosphamide[2] Cyclophosphamide ,
Cyclophosphamide 💬 		--[4] 13, 60, 96, 256 💬
44Deprakine----[1] 256 💬
45Diagnostic Test: Beck depression inventory fast screen (Questionnaire)----[3] 3, 15, 256 💬
46Diagnostic Test: Borg Scale----[3] 3, 15, 256 💬
47Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire)----[3] 3, 15, 256 💬
48Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire)----[3] 3, 15, 256 💬
49Diagnostic Test: German Pain Inventory (Questionnaire)----[3] 3, 15, 256 💬
50Diagnostic Test: Handheld Dynamometry (HHD)----[3] 3, 15, 256 💬
51Diagnostic Test: Laboratory assessment: calcium[1] Calcium---[3] 3, 15, 256 💬
52Diagnostic Test: Laboratory assessment: Creatine kinase[1] Creatine---[3] 3, 15, 256 💬
53Diagnostic Test: Laboratory assessment: magnesium[1] Magnesium---[3] 3, 15, 256 💬
54Diagnostic Test: Laboratory assessment: phosphate----[3] 3, 15, 256 💬
55Diagnostic Test: Laboratory assessment: Vitamin D Level[1] Vitamin D---[3] 3, 15, 256 💬
56Diagnostic Test: Muscle ultrasound----[5] 3, 15, 111, 113, 256 💬
57Diagnostic Test: Neutralizing Antibody to SPK-3006 capsid----[1] 256 💬
58Diagnostic Test: Pressure pain threshold----[4] 3, 15, 113, 256 💬
59Diagnostic Test: Quick Motor Function Test----[4] 3, 15, 113, 256 💬
60Diagnostic Test: R-PAct (Questionnaire)----[3] 3, 15, 256 💬
61Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire)----[3] 3, 15, 256 💬
62Diagnostic Test: Six-minute walk test (6MWT)----[3] 3, 15, 256 💬
63Diagnostic Test: Vital signs----[3] 3, 15, 256 💬
64Diphenhydramine[1] Diphenhydramine[4] Diphenhydramine ,
Diphenhydramine hydrochloride ,
Diphenhydramine salicylate ,
Diphenhydramine citrate 💬 		[1] HRH1 💬 [4] Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction 💬[12] 11, 13, 19, 49, 51, 63, 70, 86, 96, 222, 256, 300 💬
65DNL952----[1] 256 💬
66Duvoglustat[1] Duvoglustat[2] Duvoglustat ,
Duvoglustat hydrochloride 💬 		[1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
67Duvoglustat HCl[1] Duvoglustat[2] Duvoglustat ,
Duvoglustat hydrochloride 💬 		[1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
68Duvoglustat hydrochloride[1] Duvoglustat[2] Duvoglustat ,
Duvoglustat hydrochloride 💬 		[1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
69Enzyme Replacement Agent----[2] 19, 256 💬
70Filgrastim[1] Filgrastim[1] Filgrastim 💬 [1] CSF3R 💬 [5] Cytokine-cytokine receptor interaction, Hematopoietic cell lineage, JAK-STAT signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer 💬[30] 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] 256 💬
72Genetic: ACTUS-101[1] ACTUS-101---[1] 256 💬
73Genetic: Blood draw for optional genetic exome sequencing----[3] 3, 15, 256 💬
74Genetic: CRG003 injection----[1] 256 💬
75Genetic: GC301----[1] 256 💬
76Genetic: Genetic test: ACE polymorphism----[3] 3, 15, 256 💬
77Genetic: Genetic test: ACTN3 polymorphism----[3] 3, 15, 256 💬
78Genetic: Laboratory Tests----[2] 113, 256 💬
79Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase----[1] 256 💬
80Genetic: SPK-3006----[1] 256 💬
81Genetic: zocaglusagene nuzaparvovec----[1] 256 💬
82GILT-rhGAA[1] Reveglucosidase alfa---[1] 256 💬
83Glucosidase alfa----[1] 256 💬
84Glycosade----[3] 65, 256, 257 💬
85GZ402666----[1] 256 💬
86GZ419829----[1] 256 💬
87Intravenous immune globulin----[1] 256 💬
88Ketocal 4:1 liquid Nutricia (intervention)----[1] 256 💬
89LARONIDASE[1] Laronidase[1] Laronidase 💬 [1] IDUA 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[2] 19, 256 💬
90Lidocaine[1] Lidocaine[3] Lidocaine ,
Lidocaine hydrochloride ,
Lidocaine hydrochloride monohydrate 💬 		[6] SCN1A,
SCN2A,
SCN3A,
SCN5A,
SCN8A,
SCN9A 💬 		[5] Adrenergic signaling in cardiomyocytes, Cadherin signaling, Dopaminergic synapse, IgSF CAM signaling, Taste transduction 💬[14] 6, 13, 22, 34, 46, 51, 70, 86, 107, 168, 226, 231, 256, 298 💬
91LMX 4 Topical Cream----[1] 256 💬
92Low carbohydrate ketogenic diet----[1] 256 💬
93Lumizyme®----[1] 256 💬
94Magnesium[1] Magnesium---[23] 3, 6, 11, 13, 15, 46, 64, 70, 71, 93, 97, 113, 166, 225, 234, 235, 256, 284, 291, 296, 297, 298, 340 💬
95Mannose[1] Mannose[1] Demannose 💬 --[2] 13, 256 💬
96Methotrexate[1] Methotrexate[2] Methotrexate ,
Methotrexate sodium 💬 		[2] DHFR,
DHFR2 💬 		[3] Folate biosynthesis, Folate transport and metabolism, One carbon pool by folate 💬[41] 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 SODIUM[1] Methotrexate[2] Methotrexate ,
Methotrexate sodium 💬 		[2] DHFR,
DHFR2 💬 		[3] Folate biosynthesis, Folate transport and metabolism, One carbon pool by folate 💬[2] 46, 256 💬
98MIGLUSTAT[1] Miglustat[1] Miglustat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[3] 19, 256, 299 💬
99Multi-ingredient supplement (PDT-MIS)----[1] 256 💬
100MYOZYME----[1] 256 💬
101Myozyme 50 mg powder for concentrate for solution for infusion----[1] 256 💬
102MYOZYME®----[1] 256 💬
103Myozyme® (alglucosidase alfa)[1] Alglucosidase alfa[1] Alglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
104N.a.----[11] 3, 6, 8, 13, 46, 63, 96, 97, 240, 256, 271 💬
105NEO-GAA----[1] 256 💬
106NeoGAA[1] Avalglucosidase alfa[1] Avalglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
107NeoGAA or avalglucosidase alfa[1] Avalglucosidase alfa[1] Avalglucosidase alfa 💬 [1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
108Nexviadyme----[1] 256 💬
109NON APPLICABILE----[3] 49, 51, 256 💬
110Other: Blood sample----[37] 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] 256 💬
112Other: RMST----[1] 256 💬
113Other: Saline----[8] 2, 6, 46, 97, 98, 256, 274, 301 💬
114Other: Untreated----[1] 256 💬
115Pyridostigmine[1] Pyridostigmine[1] Pyridostigmine bromide 💬 [1] ACHE 💬 [3] Cholinergic synapse, Glycerophospholipid metabolism, Neuroactive ligand signaling 💬[5] 3, 6, 11, 51, 256 💬
116Pyridostigmine Bromide[1] Pyridostigmine[1] Pyridostigmine bromide 💬 [1] ACHE 💬 [3] Cholinergic synapse, Glycerophospholipid metabolism, Neuroactive ligand signaling 💬[5] 3, 6, 11, 51, 256 💬
117RAAV1-CMV-GAA (study agent) Administration----[1] 256 💬
118Rapamycin[1] Sirolimus[1] Sirolimus 💬 [1] MTOR 💬 [52] 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] 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] 256 💬
120Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)[2] Avalglucosidase alfa,
Mannose		[2] Avalglucosidase alfa ,
Demannose 💬 		[1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
121Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA)[2] Avalglucosidase alfa,
Mannose		[2] Avalglucosidase alfa ,
Demannose 💬 		[1] GAA 💬 [3] Galactose metabolism, Lysosome biogenesis, Starch and sucrose metabolism 💬[1] 256 💬
122Recombinant human acid alfa-glucosidase (rhGAA)----[1] 256 💬
123Recombinant human acid alpha-glucosidase (rhGAA)----[1] 256 💬
124RECOMBINANT HUMAN ALFA-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN----[1] 256 💬
125REN001[1] ReN001---[1] 256 💬
126Reveglucosidase alfa[1] Reveglucosidase alfa---[1] 256 💬
127Reveglucosidase alpha----[1] 256 💬
128RhGAA----[1] 256 💬
129RhGAA, acid alpha glucosidase----[1] 256 💬
130Rituxan----[5] 46, 60, 63, 256, 288 💬
131RITUXIMAB[1] Rituximab[1] Rituximab 💬 [1] MS4A1 💬 [1] Hematopoietic cell lineage 💬[54] 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] 256 💬
133Salbutamol----[9] 2, 3, 11, 85, 86, 228, 256, 299, 340 💬
134Sodium Valproate----[3] 2, 102, 256 💬
135SPK-3006----[1] 256 💬
136Ss-hydroxybuturate esters----[1] 256 💬
137Triheptanoin[1] Triheptanoin[1] Triheptanoin 💬 --[12] 2, 8, 65, 149, 156, 248, 256, 257, 317, 318, 324, 344 💬
138UX007----[5] 8, 248, 256, 257, 344 💬
139VAL-1221----[2] 256, 309 💬
140Vitamin D[1] Vitamin D---[26] 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] 19, 256, 299 💬
142Zavesca® Prescription----[1] 256 💬