| 1 | 1-deoxynojirimycin hydrochloride | [1] Duvoglustat Duvoglustat | [2] Duvoglustat
Duvoglustat
,
Duvoglustat hydrochloride
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 2 | ABX1100 injection FOR IV infusion | [1] ABX1100 ABX1100 | - | - | - | [1] 256 256 💬
|
| 3 | Acetaminophen | [1] Acetaminophen Acetaminophen | [1] Acetaminophen
Acetaminophen
💬
| [2] PTGS1 PTGS1, PTGS2 💬
| [23] Alzheimer disease 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 💬 | [14] 11 11, 19, 34, 46, 49, 51, 70, 77, 86, 96, 222, 256, 300, 337 💬
|
| 4 | Actus-101 | [1] ACTUS-101 ACTUS-101 | - | - | - | [1] 256 256 💬
|
| 5 | Albuterol | [1] Albuterol Albuterol | [2] Albuterol sulfate
Albuterol sulfate
,
Albuterol
💬
| [1] ADRB2 ADRB2 💬
| [11] Adrenergic signaling in cardiomyocytes 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 💬 | [8] 12 12, 13, 86, 113, 231, 256, 299, 340 💬
|
| 6 | Aldurazyme | [1] Laronidase Laronidase | [1] Laronidase
Laronidase
💬
| [1] IDUA IDUA 💬
| [2] Glycosaminoglycan degradation Glycosaminoglycan degradation, Lysosome 💬 | [2] 19 19, 256 💬
|
| 7 | Alglucosidase alfa | [1] Alglucosidase alfa Alglucosidase alfa | [1] Alglucosidase alfa
Alglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 8 | Alglucosidase alfa GZ419829 | [1] Alglucosidase alfa Alglucosidase alfa | [1] Alglucosidase alfa
Alglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 9 | Alglucosidase alfa or avalglucosidase alfa | [1] Alglucosidase alfa Alglucosidase alfa | [1] Alglucosidase alfa
Alglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 10 | Alglucosidase alpha | - | - | - | - | [1] 256 256 💬
|
| 11 | Alglucosidasi alfa umana ricombinante | - | - | - | - | [1] 256 256 💬
|
| 12 | Algucosidase alfa 20 MG/KG once every 4 weeks instead OF once every 2 weeks | - | - | - | - | [1] 256 256 💬
|
| 13 | Alpha-glucosidase | [1] Alglucosidase alfa Alglucosidase alfa | [1] Alglucosidase alfa
Alglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 14 | AT2220 | [1] AT2220 AT2220 | - | - | - | [1] 256 256 💬
|
| 15 | AT2221 | [1] Miglustat Miglustat | [1] Miglustat
Miglustat
💬
| [1] UGCG UGCG 💬
| [2] Cornified envelope formation Cornified envelope formation, Sphingolipid metabolism 💬 | [1] 256 256 💬
|
| 16 | AT2221 65 MG formulated capsules | [1] Miglustat Miglustat | [1] Miglustat
Miglustat
💬
| [1] UGCG UGCG 💬
| [2] Cornified envelope formation Cornified envelope formation, Sphingolipid metabolism 💬 | [1] 256 256 💬
|
| 17 | AT845 | [1] AT845 AT845 | - | - | - | [1] 256 256 💬
|
| 18 | ATB200 | - | - | - | - | [1] 256 256 💬
|
| 19 | Avalglucosidase alfa | [1] Avalglucosidase alfa Avalglucosidase alfa | [1] Avalglucosidase alfa
Avalglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 20 | Avalglucosidase ALFA-ngpt (GZ402666) IV | [1] Avalglucosidase alfa Avalglucosidase alfa | [1] Avalglucosidase alfa
Avalglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 21 | Avalglucosidasi alfa | - | - | - | - | [1] 256 256 💬
|
| 22 | Blood draw FOR optional genetic exome sequencing | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 23 | BMN 701 | - | - | - | - | [1] 256 256 💬
|
| 24 | Borg scale | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 25 | Bortezomib | [2] Bortezomib Bortezomib, Bortezomib D-mannitol | [1] Bortezomib
Bortezomib
💬
| [1] PSMB5 PSMB5 💬
| [8] Alzheimer disease Alzheimer disease, Amyotrophic lateral sclerosis, Huntington disease, Parkinson disease, Pathways of neurodegeneration - multiple diseases, Prion disease, Proteasome, Spinocerebellar ataxia 💬 | [16] 11 11, 13, 26, 28, 46, 49, 51, 61, 63, 66, 172, 222, 228, 256, 288, 331 💬
|
| 26 | Cipaglucosidase alfa | [1] Cipaglucosidase alfa Cipaglucosidase alfa | [1] Cipaglucosidase alfa
Cipaglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 27 | Cipaglucosidasi alfa | - | - | - | - | [1] 256 256 💬
|
| 28 | Clenbuterol | [1] Clenbuterol Clenbuterol | [2] Clenbuterol hydrochloride
Clenbuterol hydrochloride
,
Clenbuterol
💬
| [1] ADRB2 ADRB2 💬
| [11] Adrenergic signaling in cardiomyocytes 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 💬 | [4] 1 1, 2, 113, 256 💬
|
| 29 | CRG003 injection | - | - | - | - | [1] 256 256 💬
|
| 30 | Cyclophosphamide monohydrate | [1] Cyclophosphamide Cyclophosphamide | [2] Cyclophosphamide
Cyclophosphamide
,
Cyclophosphamide
💬
| - | - | [4] 13 13, 60, 96, 256 💬
|
| 31 | Deprakine | - | - | - | - | [1] 256 256 💬
|
| 32 | Diphenhydramine | [1] Diphenhydramine Diphenhydramine | [4] Diphenhydramine
Diphenhydramine
,
Diphenhydramine hydrochloride
,
Diphenhydramine salicylate
,
Diphenhydramine citrate
💬
| [1] HRH1 HRH1 💬
| [4] Calcium signaling pathway Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction 💬 | [10] 13 13, 19, 49, 51, 70, 86, 96, 222, 256, 300 💬
|
| 33 | Duvoglustat | [1] Duvoglustat Duvoglustat | [2] Duvoglustat
Duvoglustat
,
Duvoglustat hydrochloride
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 34 | Duvoglustat HCL | [1] Duvoglustat Duvoglustat | [2] Duvoglustat
Duvoglustat
,
Duvoglustat hydrochloride
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 35 | Duvoglustat hydrochloride | [1] Duvoglustat Duvoglustat | [2] Duvoglustat
Duvoglustat
,
Duvoglustat hydrochloride
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 36 | Enzyme replacement agent | - | - | - | - | [2] 19 19, 256 💬
|
| 37 | Filgrastim | [1] Filgrastim Filgrastim | [1] Filgrastim
Filgrastim
💬
| [1] CSF3R CSF3R 💬
| [5] Cytokine-cytokine receptor interaction Cytokine-cytokine receptor interaction, Hematopoietic cell lineage, JAK-STAT signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer 💬 | [29] 2 2, 6, 13, 25, 26, 28, 34, 35, 38, 40, 41, 42, 45, 46, 47, 49, 51, 60, 62, 65, 96, 113, 164, 256, 283, 284, 285, 296, 331 💬
|
| 38 | GC301 | - | - | - | - | [1] 256 256 💬
|
| 39 | Genetic test: ACE polymorphism | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 40 | Genetic test: ACTN3 polymorphism | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 41 | GILT-rhgaa | [1] Reveglucosidase alfa Reveglucosidase alfa | - | - | - | [1] 256 256 💬
|
| 42 | Glucosidase alfa | - | - | - | - | [1] 256 256 💬
|
| 43 | Glycosade | - | - | - | - | [3] 65 65, 256, 257 💬
|
| 44 | GZ402666 | - | - | - | - | [1] 256 256 💬
|
| 45 | GZ419829 | - | - | - | - | [1] 256 256 💬
|
| 46 | Intravenous immune globulin | - | - | - | - | [1] 256 256 💬
|
| 47 | Laboratory assessment: calcium | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 48 | Laboratory assessment: creatine kinase | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 49 | Laboratory assessment: magnesium | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 50 | Laboratory assessment: phosphate | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 51 | Laboratory assessment: vitamin D level | - | - | - | - | [3] 3 3, 15, 256 💬
|
| 52 | Laboratory tests | - | - | - | - | [3] 19 19, 113, 256 💬
|
| 53 | Laronidase | [1] Laronidase Laronidase | [1] Laronidase
Laronidase
💬
| [1] IDUA IDUA 💬
| [2] Glycosaminoglycan degradation Glycosaminoglycan degradation, Lysosome 💬 | [2] 19 19, 256 💬
|
| 54 | Lidocaine | [1] Lidocaine Lidocaine | [3] Lidocaine
Lidocaine
,
Lidocaine hydrochloride
,
Lidocaine hydrochloride monohydrate
💬
| [5] SCN1A SCN1A, SCN2A, SCN3A, SCN5A, SCN9A 💬
| [3] Adrenergic signaling in cardiomyocytes Adrenergic signaling in cardiomyocytes, Dopaminergic synapse, Taste transduction 💬 | [10] 6 6, 13, 22, 46, 86, 107, 168, 226, 231, 256 💬
|
| 55 | LMX 4 topical cream | - | - | - | - | [1] 256 256 💬
|
| 56 | LOW carbohydrate ketogenic diet | - | - | - | - | [1] 256 256 💬
|
| 57 | Mannose | [1] Mannose Mannose | - | - | - | [1] 256 256 💬
|
| 58 | Methotrexate | [1] Methotrexate Methotrexate | [2] Methotrexate
Methotrexate
,
Methotrexate sodium
💬
| [2] DHFR DHFR, DHFR 💬
| [3] Folate biosynthesis Folate biosynthesis, Folate transport and metabolism, One carbon pool by folate 💬 | [37] 11 11, 13, 19, 34, 35, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 53, 54, 55, 60, 62, 65, 84, 93, 96, 97, 107, 162, 164, 172, 256, 270, 271, 284, 285, 286, 326, 331 💬
|
| 59 | Methotrexate sodium | [1] Methotrexate Methotrexate | [2] Methotrexate
Methotrexate
,
Methotrexate sodium
💬
| [2] DHFR DHFR, DHFR 💬
| [3] Folate biosynthesis Folate biosynthesis, Folate transport and metabolism, One carbon pool by folate 💬 | [2] 46 46, 256 💬
|
| 60 | Miglustat | [1] Miglustat Miglustat | [1] Miglustat
Miglustat
💬
| [1] UGCG UGCG 💬
| [2] Cornified envelope formation Cornified envelope formation, Sphingolipid metabolism 💬 | [3] 19 19, 256, 299 💬
|
| 61 | Muscle ultrasound | - | - | - | - | [4] 3 3, 15, 113, 256 💬
|
| 62 | Myozyme | [1] Alglucosidase alfa Alglucosidase alfa | [1] Alglucosidase alfa
Alglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 63 | Myozyme 50 MG powder FOR concentrate FOR solution FOR infusion | [1] Alglucosidase alfa Alglucosidase alfa | [1] Alglucosidase alfa
Alglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 64 | NEO-GAA | - | - | - | - | [1] 256 256 💬
|
| 65 | Neogaa | [1] Avalglucosidase alfa Avalglucosidase alfa | [1] Avalglucosidase alfa
Avalglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 66 | Neogaa or avalglucosidase alfa | [1] Avalglucosidase alfa Avalglucosidase alfa | [1] Avalglucosidase alfa
Avalglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 67 | Neutralizing antibody TO SPK-3006 capsid | - | - | - | - | [1] 256 256 💬
|
| 68 | Nexviadyme | [1] Avalglucosidase alfa Avalglucosidase alfa | [1] Avalglucosidase alfa
Avalglucosidase alfa
💬
| [1] GAA GAA 💬
| [3] Galactose metabolism Galactose metabolism, Lysosome, Starch and sucrose metabolism 💬 | [1] 256 256 💬
|
| 69 | NON applicabile | - | - | - | - | [3] 49 49, 51, 256 💬
|
| 70 | Phosphate | [1] Phosphate ion Phosphate ion | - | - | - | [13] 3 3, 11, 13, 46, 65, 70, 75, 90, 235, 238, 256, 286, 288 💬
|
| 71 | Pressure pain threshold | - | - | - | - | [4] 3 3, 15, 113, 256 💬
|
| 72 | Pyridostigmine bromide | [1] Pyridostigmine Pyridostigmine | [1] Pyridostigmine bromide
Pyridostigmine bromide
💬
| [1] ACHE ACHE 💬
| [3] Cholinergic synapse Cholinergic synapse, Glycerophospholipid metabolism, Neuroactive ligand signaling 💬 | [5] 3 3, 6, 11, 51, 256 💬
|
| 73 | Quick motor function test | - | - | - | - | [4] 3 3, 15, 113, 256 💬
|
| 74 | RAAV1-CMV-GAA (study agent) administration | - | - | - | - | [1] 256 256 💬
|
| 75 | Rapamycin | [1] Sirolimus Sirolimus | [1] Sirolimus
Sirolimus
💬
| [1] MTOR MTOR 💬
| [49] AMPK signaling pathway 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, 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 💬 | [19] 2 2, 15, 34, 35, 46, 49, 51, 53, 60, 61, 63, 67, 86, 96, 158, 193, 256, 279, 283 💬
|
| 76 | Recombinant adeno-associated virus acid alpha-glucosidase | - | - | - | - | [1] 256 256 💬
|
| 77 | Recombinant human ?-glucosidase conjugated with synthetic bismannose-6-phosphate-MAN6 glycan | - | - | - | - | [1] 256 256 💬
|
| 78 | Recombinant human ALFA-glucosidase conjugated with synthetic bismannose-6-phosphate-MAN6 glycan | - | - | - | - | [1] 256 256 💬
|
| 79 | REN001 | [1] ReN001 ReN001 | - | - | - | [1] 256 256 💬
|
| 80 | Reveglucosidase alfa | [1] Reveglucosidase alfa Reveglucosidase alfa | - | - | - | [1] 256 256 💬
|
| 81 | Reveglucosidase alpha | - | - | - | - | [1] 256 256 💬
|
| 82 | Rhgaa | - | - | - | - | [1] 256 256 💬
|
| 83 | Rhgaa, acid alpha glucosidase | - | - | - | - | [1] 256 256 💬
|
| 84 | Rituxan | [1] Rituximab Rituximab | [1] Rituximab
Rituximab
💬
| [1] MS4A1 MS4A1 💬
| [1] Hematopoietic cell lineage Hematopoietic cell lineage 💬 | [7] 35 35, 46, 60, 61, 63, 256, 288 💬
|
| 85 | Rituximab | [1] Rituximab Rituximab | [1] Rituximab
Rituximab
💬
| [1] MS4A1 MS4A1 💬
| [1] Hematopoietic cell lineage Hematopoietic cell lineage 💬 | [52] 11 11, 13, 14, 19, 26, 34, 35, 36, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 53, 55, 56, 60, 61, 62, 63, 64, 65, 66, 83, 84, 85, 86, 93, 94, 96, 97, 162, 172, 220, 222, 229, 256, 271, 274, 283, 284, 285, 288, 296, 300, 331 💬
|
| 86 | Rmst | - | - | - | - | [1] 256 256 💬
|
| 87 | Salbutamol | [1] Albuterol Albuterol | [2] Albuterol sulfate
Albuterol sulfate
,
Albuterol
💬
| [1] ADRB2 ADRB2 💬
| [11] Adrenergic signaling in cardiomyocytes 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] 2 2, 3, 85, 86, 228, 256, 273, 299, 340 💬
|
| 88 | Saline | [1] Sodium chloride Sodium chloride | [1] Sodium chloride
Sodium chloride
💬
| - | - | [19] 2 2, 6, 13, 17, 18, 46, 53, 67, 77, 86, 96, 97, 98, 168, 256, 271, 274, 299, 301 💬
|
| 89 | Sodium valproate | - | - | - | - | [3] 2 2, 102, 256 💬
|
| 90 | SPK-3006 | [1] Vanglusagene ensiparvovec Vanglusagene ensiparvovec | - | - | - | [1] 256 256 💬
|
| 91 | Triheptanoin | [1] Triheptanoin Triheptanoin | [1] Triheptanoin
Triheptanoin
💬
| - | - | [10] 2 2, 8, 65, 149, 156, 248, 256, 257, 317, 324 💬
|
| 92 | UX007 | - | - | - | - | [5] 8 8, 248, 256, 257, 344 💬
|
| 93 | VAL-1221 | - | - | - | - | [2] 256 256, 309 💬
|
| 94 | Vital signs | - | - | - | - | [4] 3 3, 15, 19, 256 💬
|
| 95 | Zavesca | [1] Miglustat Miglustat | [1] Miglustat
Miglustat
💬
| [1] UGCG UGCG 💬
| [2] Cornified envelope formation Cornified envelope formation, Sphingolipid metabolism 💬 | [3] 19 19, 256, 299 💬
|
| 96 | Zavesca® prescription | - | - | - | - | [1] 256 256 💬
|
| 97 | Zocaglusagene nuzaparvovec | - | - | - | - | [1] 256 256 💬
|