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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
1045251016----1件: 19
21- Deoxygalactonojirimycin----1件: 19
31-Deoxygalactonojirimycin1件: Migalastat1件: D10359 --1件: 19
41-deoxygalactonojirimycin hydrochloride1件: Migalastat1件: D10359 --1件: 19
512 lead electrocardiogram----1件: 19
612S-IT----1件: 19
715 O Water1件: Water1件: D00001 --1件: 19
82(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID----2件: 19, 65
92-aminoethanethiol1件: Cysteamine2件: D03634 ,
D03635 		--2件: 19, 299
102-hour Holter Monitor1件: Methamidophos---1件: 19
112-HYDROXYPROPYL-BETA-CYCLODEXTRIN2件: Betadex,
Hydroxypropyl betadex		1件: D02401 --1件: 19
124D-310----1件: 19
13500 0500 0----1件: 19
1489 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)----1件: 19
1589Zr-SBC-103----1件: 19
16[14C] AT1001----1件: 19
17A (migalastat)1件: Migalastat1件: D10359 --1件: 19
18A16AB04----1件: 19
19A16AX06----1件: 19
20AAV2/8.TBG.hARSB----1件: 19
21AAV2CUhCLN2 (3x10^12 particle units)----1件: 19
22AAV9-CAG-coh-SGSH----1件: 19
23AAV9-GLB1----1件: 19
24AAVrh.10-hMPSIIIA----1件: 19
25AAVrh.10cuARSA----1件: 19
26AAVrh.10CUCLN2----1件: 19
27AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies----1件: 19
28AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies----1件: 19
29Abatacept1件: Abatacept1件: D03203 2件: CD80,
CD86 💬		12件: Allograft rejection, Autoimmune thyroid disease, Cell adhesion molecules, Graft-versus-host disease, Intestinal immune network for IgA production, Kaposi sarcoma-associated herpesvirus infection, Rheumatoid arthritis, Systemic lupus erythematosus, Toll-like receptor signaling pathway, Transcriptional misregulation in cancer, Type I diabetes mellitus, Viral myocarditis 💬26件: 11, 13, 19, 41, 44, 46, 49, 50, 51, 53, 55, 60, 65, 84, 93, 95, 96, 97, 107, 160, 164, 222, 271, 284, 285, 300
30Abcertin----1件: 19
31ABO-101----1件: 19
32ABO-102----1件: 19
33Accupro----1件: 19
34Accuretic1件: Quinapril2件: D00459 ,
D03752 		1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬1件: 19
35Acetylcysteine1件: Acetylcysteine1件: D00221 --20件: 6, 13, 19, 20, 34, 49, 51, 53, 58, 85, 90, 94, 111, 164, 193, 233, 296, 298, 299, 337
36ACT-434964----1件: 19
37Adalimumab1件: Adalimumab1件: D02597 1件: TNF 💬67件: AGE-RAGE signaling pathway in diabetic complications, Adipocytokine signaling pathway, African trypanosomiasis, Alcoholic liver disease, Allograft rejection, Alzheimer disease, Amoebiasis, Amyotrophic lateral sclerosis, Antifolate resistance, Antigen processing and presentation, Apoptosis, Asthma, C-type lectin receptor signaling pathway, Chagas disease, Coronavirus disease - COVID-19, Cytokine-cytokine receptor interaction, Dilated cardiomyopathy, Epstein-Barr virus infection, Fc epsilon RI signaling pathway, Fluid shear stress and atherosclerosis, Graft-versus-host disease, Hematopoietic cell lineage, Hepatitis B, Hepatitis C, Herpes simplex virus 1 infection, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Human papillomavirus infection, Hypertrophic cardiomyopathy, IL-17 signaling pathway, Inflammatory bowel disease, Influenza A, Insulin resistance, Legionellosis, Leishmaniasis, Lipid and atherosclerosis, MAPK signaling pathway, Malaria, NF-kappa B signaling pathway, NOD-like receptor signaling pathway, Natural killer cell mediated cytotoxicity, Necroptosis, Non-alcoholic fatty liver disease, Osteoclast differentiation, Pathogenic Escherichia coli infection, Pathways of neurodegeneration - multiple diseases, Pertussis, Prion disease, Proteoglycans in cancer, RIG-I-like receptor signaling pathway, Rheumatoid arthritis, Salmonella infection, Shigellosis, Sphingolipid signaling pathway, Systemic lupus erythematosus, T cell receptor signaling pathway, TGF-beta signaling pathway, TNF signaling pathway, Toll-like receptor signaling pathway, Toxoplasmosis, Tuberculosis, Type I diabetes mellitus, Type II diabetes mellitus, Viral protein interaction with cytokine and cytokine receptor, Yersinia infection, mTOR signaling pathway 💬18件: 19, 37, 40, 41, 46, 56, 84, 96, 97, 107, 151, 160, 164, 222, 226, 269, 270, 271
38Adalimumab Injection [Humira]1件: Adalimumab1件: D02597 1件: TNF 💬67件: AGE-RAGE signaling pathway in diabetic complications, Adipocytokine signaling pathway, African trypanosomiasis, Alcoholic liver disease, Allograft rejection, Alzheimer disease, Amoebiasis, Amyotrophic lateral sclerosis, Antifolate resistance, Antigen processing and presentation, Apoptosis, Asthma, C-type lectin receptor signaling pathway, Chagas disease, Coronavirus disease - COVID-19, Cytokine-cytokine receptor interaction, Dilated cardiomyopathy, Epstein-Barr virus infection, Fc epsilon RI signaling pathway, Fluid shear stress and atherosclerosis, Graft-versus-host disease, Hematopoietic cell lineage, Hepatitis B, Hepatitis C, Herpes simplex virus 1 infection, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Human papillomavirus infection, Hypertrophic cardiomyopathy, IL-17 signaling pathway, Inflammatory bowel disease, Influenza A, Insulin resistance, Legionellosis, Leishmaniasis, Lipid and atherosclerosis, MAPK signaling pathway, Malaria, NF-kappa B signaling pathway, NOD-like receptor signaling pathway, Natural killer cell mediated cytotoxicity, Necroptosis, Non-alcoholic fatty liver disease, Osteoclast differentiation, Pathogenic Escherichia coli infection, Pathways of neurodegeneration - multiple diseases, Pertussis, Prion disease, Proteoglycans in cancer, RIG-I-like receptor signaling pathway, Rheumatoid arthritis, Salmonella infection, Shigellosis, Sphingolipid signaling pathway, Systemic lupus erythematosus, T cell receptor signaling pathway, TGF-beta signaling pathway, TNF signaling pathway, Toll-like receptor signaling pathway, Toxoplasmosis, Tuberculosis, Type I diabetes mellitus, Type II diabetes mellitus, Viral protein interaction with cytokine and cytokine receptor, Yersinia infection, mTOR signaling pathway 💬1件: 19
39ADENO-ASSOCIATED VIRAL (AAV) SEROTYPE 8 (AAV2/8) VECTOR WITH LIVER-SPECIFIC THYROXINE-BINDING GLOBULIN (TBG) PROMOTER, DRIVING THE EXPRESSION OF THE HUMAN ARSB GENE1件: Levothyroxine2件: D01010 ,
D08125 		2件: THRA,
THRB 💬		2件: Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬1件: 19
40Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene----1件: 19
41Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-betagal)----1件: 19
42Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal)----1件: 19
43Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
44Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
45Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector----1件: 19
46Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector----1件: 19
47Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector----1件: 19
48Adeno-associated virus serotype hu68 containing the human GALC gene----1件: 19
49Adeno-associated virus serotype rh.10 expressing beta-galactosidase1件: Tilactase1件: D03348 --1件: 19
50ADMINISTRATION----1件: 19
51Administration Kit----1件: 19
52Adrabetadex1件: Adrabetadex---1件: 19
53Afegostat1件: Afegostat1件: D09576 --1件: 19
54Afegostat tartrate1件: Afegostat1件: D09576 --1件: 19
55Agalsidase----1件: 19
56AGALSIDASE ALFA1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
57Agalsidase Alpha1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
58AGALSIDASE BETA1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
59Agalsidase beta (GZ419828)1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
60Agalsidase beta (recombinant form)1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
61AGALSIDASI ALFA----1件: 19
62AGALSIDASI BETA----1件: 19
63AGT-181----1件: 19
64AGT-181 (HIRMAb-IDUA)----1件: 19
65AGT-182----1件: 19
66Aldurazyme1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
67Aldurazyme (laronidase)1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬2件: 19, 256
68Aldurazyme (Recombinant Human Alpha-L-Iduronidase)1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
69ALDURAZYME*INFUS 1FL 5ML 500U1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
70ALEMTUZUMAB1件: Alemtuzumab1件: D02802 1件: CD52 💬-17件: 13, 15, 19, 20, 36, 43, 44, 46, 51, 60, 63, 65, 85, 96, 164, 283, 284
71Alendronate1件: Alendronic acid2件: D00939 ,
D07119 		1件: FDPS 💬4件: Human T-cell leukemia virus 1 infection, Influenza A, Metabolic pathways, Terpenoid backbone biosynthesis 💬7件: 19, 41, 46, 50, 271, 274, 299
72Alendronate sodium1件: Alendronic acid2件: D00939 ,
D07119 		1件: FDPS 💬4件: Human T-cell leukemia virus 1 infection, Influenza A, Metabolic pathways, Terpenoid backbone biosynthesis 💬2件: 19, 299
73Alfa-manosidadas humana recombinante----1件: 19
74Allogeneic stem cell transplantation----5件: 19, 20, 49, 62, 84
75Alpha-galactosidase A1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
76Ambroxol1件: Ambroxol1件: D07442 --2件: 6, 19
77Anakinra1件: Anakinra1件: D02934 2件: IL1R1,
IL1R2 💬		15件: Amoebiasis, Cytokine-cytokine receptor interaction, Fluid shear stress and atherosclerosis, Hematopoietic cell lineage, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Inflammatory mediator regulation of TRP channels, MAPK signaling pathway, NF-kappa B signaling pathway, Osteoclast differentiation, Pathogenic Escherichia coli infection, Prostate cancer, Shigellosis, Th17 cell differentiation, Transcriptional misregulation in cancer 💬17件: 2, 13, 15, 19, 28, 37, 41, 46, 49, 50, 56, 84, 86, 97, 106, 107, 299
78Angiotensin1件: Angiotensin II2件: D00150 ,
D02014 		2件: AGTR1,
AGTR2 💬		16件: AGE-RAGE signaling pathway in diabetic complications, Adrenergic signaling in cardiomyocytes, Aldosterone synthesis and secretion, Apelin signaling pathway, Calcium signaling pathway, Coronavirus disease - COVID-19, Cortisol synthesis and secretion, Cushing syndrome, Diabetic cardiomyopathy, Neuroactive ligand-receptor interaction, Pathways in cancer, Phospholipase D signaling pathway, Renin secretion, Renin-angiotensin system, Vascular smooth muscle contraction, cGMP-PKG signaling pathway 💬7件: 17, 19, 46, 66, 86, 222, 224
79ARIMOCLOMOL1件: Arimoclomol1件: D11374 --3件: 2, 15, 19
80Arimoclomol (hard gelatine capsule)2件: Arimoclomol,
Gelatin		3件: D00115 ,
D06892 ,
D11374 		--1件: 19
81Arimoclomol (HPMC capsule)1件: Arimoclomol1件: D11374 --1件: 19
82Arimoclomol citrate1件: Arimoclomol1件: D11374 --2件: 15, 19
83Arylsulfatase A----1件: 19
84AT-GTX-501----1件: 19
85AT1001----1件: 19
86AT1001 150 mg----1件: 19
87AT2101----1件: 19
88ATALUREN1件: Ataluren1件: D09323 --6件: 19, 113, 140, 246, 299, 329
89AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE----1件: 19
90Autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA----1件: 19
91Autologous Plasmablasts----1件: 19
92AVR-RD-01----1件: 19
93AVR-RD-02----1件: 19
94AX----1件: 19
95AX 250----1件: 19
96AXO-AAV-GM2 High Dose----1件: 19
97AXO-AAV-GM2 Low Dose----1件: 19
98AXO-AAV-GM2 Middle Dose----1件: 19
99AXO-AAV-GM2 Starting Dose----1件: 19
100Azathioprine1件: Azathioprine2件: D00238 ,
D03033 		--21件: 6, 11, 13, 19, 35, 42, 43, 44, 45, 46, 49, 63, 65, 66, 85, 93, 94, 95, 96, 97, 162
101Azathioprine (AZA)1件: Azathioprine2件: D00238 ,
D03033 		--3件: 13, 19, 97
102B (migalastat)1件: Migalastat1件: D10359 --1件: 19
103Barium1件: Barium---3件: 2, 19, 98
104Behavioral: Neurocognitive testing----1件: 19
105Behavioral: PICC line placement----1件: 19
106Beta-cyclodextrin1件: Betadex1件: D02401 --1件: 19
107Beta-Galactosidase1件: Tilactase1件: D03348 --1件: 19
108Beta-Mercaptoethylamine1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
109Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
110BHP001----1件: 19
111Biological assays----1件: 19
112Biopsy----4件: 19, 51, 78, 193
113Blood draw----5件: 6, 19, 65, 231, 299
114Blood sampling----6件: 13, 17, 19, 46, 51, 96
115BM110----1件: 19
116BMN 110----1件: 19
117BMN 110 - Every Other Week----1件: 19
118BMN 110 - Weekly----1件: 19
119BMN 110 Every Other Week----1件: 19
120BMN 110 Weekly----1件: 19
121BMN 190----1件: 19
122BMN 250----1件: 19
123BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)----1件: 19
124Brain MRI/MRS/fMRI----1件: 19
125Brineura1件: Cerliponase alfa1件: D10813 1件: TPP1 💬1件: Lysosome 💬1件: 19
126BUSILVEX1件: Busulfan1件: D00248 --4件: 19, 36, 60, 65
127BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI1件: Busulfan1件: D00248 --2件: 19, 65
128Busulfan1件: Busulfan1件: D00248 --13件: 19, 20, 28, 36, 49, 51, 60, 65, 164, 274, 284, 285, 326
129Busulfan, Cyclophosphamide, Antithymocyte Globulin2件: Busulfan,
Cyclophosphamide		3件: D00248 ,
D00287 ,
D07760 		--2件: 19, 20
130Busulfan, Cyclophosphamide, ATG2件: Busulfan,
Cyclophosphamide		3件: D00248 ,
D00287 ,
D07760 		--1件: 19
131BUSULFANO1件: Busulfan1件: D00248 --1件: 19
132C (migalastat)1件: Migalastat1件: D10359 --1件: 19
133Calcium1件: Calcium---30件: 3, 6, 13, 15, 19, 46, 49, 51, 53, 56, 68, 75, 79, 84, 93, 94, 95, 96, 97, 186, 214, 215, 222, 224, 235, 256, 265, 274, 298, 299
134Calcium carbonate3件: Calcium,
Calcium carbonate,
Carbonate ion		2件: D00932 ,
D10802 		--8件: 13, 19, 46, 53, 75, 96, 235, 299
135CALD HR-D (High-Risk, Regimen C)----5件: 19, 20, 125, 234, 326
136CALD HR-D (High-Risk, Regimen D)----5件: 19, 20, 125, 234, 326
137CALD SR-A (Standard-Risk, Regimen A)----5件: 19, 20, 125, 234, 326
138CALD SR-B (Standard-Risk, Regimen B)----5件: 19, 20, 125, 234, 326
139Campath1件: Alemtuzumab1件: D02802 1件: CD52 💬-12件: 19, 20, 49, 51, 60, 62, 65, 164, 234, 283, 285, 326
140Campath-1H1件: Alemtuzumab1件: D02802 1件: CD52 💬-6件: 19, 20, 60, 234, 285, 326
141Cannabis1件: Medical Cannabis---9件: 2, 6, 13, 19, 46, 90, 96, 97, 271
142Cannabis sativa L., folium cum flore1件: Medical Cannabis---1件: 19
143Capsaicin1件: Capsaicin1件: D00250 1件: TRPV1 💬2件: Inflammatory mediator regulation of TRP channels, Neuroactive ligand-receptor interaction 💬5件: 2, 6, 19, 86, 226
144Carbonate1件: Carbonate ion---12件: 2, 13, 17, 18, 19, 46, 53, 75, 96, 127, 235, 299
145Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi----1件: 19
146Cerdelga1件: Eliglustat1件: D09893 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
147Ceredase1件: Alglucerase1件: D02810 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
148CEREDASE™1件: Alglucerase1件: D02810 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
149CEREZYME1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
150Cerezyme (imiglucerase for injection)1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
151Cerezyme 400 Units Powder for concentrate for solution for infusion1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
152Cerezyme®1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
153Cerezyme® / Imiglucerase1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
154Cerliponase Alfa1件: Cerliponase alfa1件: D10813 1件: TPP1 💬1件: Lysosome 💬1件: 19
155CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE----1件: 19
156CHOLECALCIFEROL1件: Cholecalciferol1件: D00188 1件: VDR 💬5件: Chemical carcinogenesis - receptor activation, Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis 💬10件: 13, 19, 34, 46, 49, 75, 96, 97, 298, 299
157Cholesterol1件: Cholesterol1件: D00040 --6件: 19, 90, 160, 259, 261, 310
158Clofarabine1件: Clofarabine1件: D03546 3件: POLA1,
POLA2,
RRM1 💬		6件: DNA replication, Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬9件: 19, 20, 40, 46, 49, 60, 96, 234, 326
159Contrast sensitivity measurement----1件: 19
160Coversyl1件: Perindopril2件: D00624 ,
D03753 		1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬2件: 19, 167
161Coversyl PLUS----1件: 19
162CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE----1件: 19
163CsA----8件: 19, 49, 50, 53, 60, 63, 222, 228
164Cyclophosphamide1件: Cyclophosphamide2件: D00287 ,
D07760 		--45件: 11, 13, 14, 16, 19, 20, 26, 28, 35, 36, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 56, 60, 62, 65, 66, 84, 85, 93, 96, 162, 164, 172, 205, 222, 224, 256, 265, 274, 283, 284, 285, 288, 326, 331
165Cyclosporine1件: Cyclosporine1件: D00184 5件: PPP3CA,
PPP3CB,
PPP3CC,
PPP3R1,
PPP3R2 💬		33件: Alzheimer disease, Amphetamine addiction, Amyotrophic lateral sclerosis, Axon guidance, B cell receptor signaling pathway, C-type lectin receptor signaling pathway, Calcium signaling pathway, Cellular senescence, Dopaminergic synapse, Glucagon signaling pathway, Glutamatergic synapse, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Kaposi sarcoma-associated herpesvirus infection, Lipid and atherosclerosis, Long-term potentiation, MAPK signaling pathway, Natural killer cell mediated cytotoxicity, Oocyte meiosis, Osteoclast differentiation, Oxytocin signaling pathway, PD-L1 expression and PD-1 checkpoint pathway in cancer, Pathways of neurodegeneration - multiple diseases, Prion disease, Renin secretion, T cell receptor signaling pathway, Th1 and Th2 cell differentiation, Th17 cell differentiation, Tuberculosis, VEGF signaling pathway, Wnt signaling pathway, cGMP-PKG signaling pathway 💬37件: 11, 19, 20, 28, 36, 38, 39, 40, 41, 42, 45, 46, 49, 50, 53, 56, 58, 60, 61, 62, 63, 64, 65, 90, 95, 97, 107, 162, 164, 222, 226, 228, 234, 283, 284, 285, 302
166Cyclosporine A1件: Cyclosporine1件: D00184 5件: PPP3CA,
PPP3CB,
PPP3CC,
PPP3R1,
PPP3R2 💬		33件: Alzheimer disease, Amphetamine addiction, Amyotrophic lateral sclerosis, Axon guidance, B cell receptor signaling pathway, C-type lectin receptor signaling pathway, Calcium signaling pathway, Cellular senescence, Dopaminergic synapse, Glucagon signaling pathway, Glutamatergic synapse, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Kaposi sarcoma-associated herpesvirus infection, Lipid and atherosclerosis, Long-term potentiation, MAPK signaling pathway, Natural killer cell mediated cytotoxicity, Oocyte meiosis, Osteoclast differentiation, Oxytocin signaling pathway, PD-L1 expression and PD-1 checkpoint pathway in cancer, Pathways of neurodegeneration - multiple diseases, Prion disease, Renin secretion, T cell receptor signaling pathway, Th1 and Th2 cell differentiation, Th17 cell differentiation, Tuberculosis, VEGF signaling pathway, Wnt signaling pathway, cGMP-PKG signaling pathway 💬11件: 19, 20, 36, 49, 50, 53, 58, 60, 107, 234, 283
167Cyclosporine A (CsA)1件: Cyclosporine1件: D00184 5件: PPP3CA,
PPP3CB,
PPP3CC,
PPP3R1,
PPP3R2 💬		33件: Alzheimer disease, Amphetamine addiction, Amyotrophic lateral sclerosis, Axon guidance, B cell receptor signaling pathway, C-type lectin receptor signaling pathway, Calcium signaling pathway, Cellular senescence, Dopaminergic synapse, Glucagon signaling pathway, Glutamatergic synapse, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Kaposi sarcoma-associated herpesvirus infection, Lipid and atherosclerosis, Long-term potentiation, MAPK signaling pathway, Natural killer cell mediated cytotoxicity, Oocyte meiosis, Osteoclast differentiation, Oxytocin signaling pathway, PD-L1 expression and PD-1 checkpoint pathway in cancer, Pathways of neurodegeneration - multiple diseases, Prion disease, Renin secretion, T cell receptor signaling pathway, Th1 and Th2 cell differentiation, Th17 cell differentiation, Tuberculosis, VEGF signaling pathway, Wnt signaling pathway, cGMP-PKG signaling pathway 💬2件: 19, 60
168Cystadane anhydrous----1件: 19
169CYSTADROPS1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
170CYSTADROPS 0.55% eye drops, solution1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
171CYSTAGON1件: Cysteamine2件: D03634 ,
D03635 		--3件: 8, 19, 299
172Cystagon 150mg1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
173Cystagon 50mg1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
174Cystagon®1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
175Cystagon® (Cysteamine Bitartrate)1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
176Cysteamine1件: Cysteamine2件: D03634 ,
D03635 		--4件: 8, 19, 21, 299
177Cysteamine (mercaptamine) viscous solution1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
178Cysteamine Bitartrate1件: Cysteamine2件: D03634 ,
D03635 		--4件: 8, 19, 21, 299
179Cysteamine bitartrate (INN: mercaptamine bitartrate1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
180Cysteamine bitartrate (INN: mercaptamine bitartrate )1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
181Cysteamine bitartrate (INN: mercaptamine bitartrate)1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
182Cysteamine Bitartrate Delayed-release Capsules1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
183Cysteamine Bitartrate Delayed-release Capsules (RP103)1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
184CYSTEAMINE HYDROCHLORIDE1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
185Cysteine1件: Cysteine2件: D00026 ,
D02326 		--11件: 6, 13, 19, 34, 84, 85, 88, 90, 254, 296, 310
186D (migalastat)1件: Migalastat1件: D10359 --1件: 19
187DEXA scan----3件: 19, 46, 96
188Diagnostic Test: Audiology assessmentwith ABR----1件: 19
189Diagnostic Test: Bone density scan (DEXA----1件: 19
190Diagnostic Test: Echocardiogram----1件: 19
191Diagnostic Test: Echocardiography at M24----1件: 19
192Diagnostic Test: Echocardiography at T0----1件: 19
193Diagnostic Test: Electrocardiogram (EKG)----1件: 19
194Diagnostic Test: Exercise test----1件: 19
195Diagnostic Test: Laboratory tests----1件: 19
196Diagnostic Test: Plasma a-Gal A activity----1件: 19
197DNL310----1件: 19
198DNL310 Drug Substance----1件: 19
199Dried blood spot (DBS) sampling----1件: 19
200DRX005B----1件: 19
201DUOC-01----3件: 13, 19, 20
202E (migalastat)1件: Migalastat1件: D10359 --1件: 19
203Echocardiogram----2件: 19, 215
204Elaprase1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
205Elaprase 2mg/ml1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
206Elaprase for intravenous (IV) infusion1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
207Elaprase_0.5mg/kg----1件: 19
208Elelyso1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
209Elelyso 60 units/kg1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
210Eliglustat1件: Eliglustat1件: D09893 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
211Eliglustat GZ3856601件: Eliglustat1件: D09893 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
212ELIGLUSTAT TARTRATE1件: Eliglustat1件: D09893 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
213Eliglustat Tartrate – Powder for Oral Suspension1件: Eliglustat1件: D09893 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
214Elosulfase alfa1件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
215Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]1件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
216ELX-021件: ELX-02---2件: 19, 299
217Enalapril1件: Enalapril2件: D00621 ,
D07892 		1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬6件: 19, 46, 66, 113, 210, 222
218Enalapril and other angiotensin converting enzyme inhibitors2件: Angiotensin II,
Enalapril		4件: D00150 ,
D00621 ,
D02014 ,
D07892 		3件: ACE,
AGTR1,
AGTR2 💬		18件: AGE-RAGE signaling pathway in diabetic complications, Adrenergic signaling in cardiomyocytes, Aldosterone synthesis and secretion, Apelin signaling pathway, Calcium signaling pathway, Chagas disease, Coronavirus disease - COVID-19, Cortisol synthesis and secretion, Cushing syndrome, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Neuroactive ligand-receptor interaction, Pathways in cancer, Phospholipase D signaling pathway, Renin secretion, Renin-angiotensin system, Vascular smooth muscle contraction, cGMP-PKG signaling pathway 💬1件: 19
219Enriched Hematopoetic Stem Cell Infusion----3件: 13, 19, 284
220Enzyme replacement----1件: 19
221Enzyme replacement therapy----1件: 19
222Enzyme replacement therapy (Agalsidase alpha (Replagal®))1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
223Enzyme replacement therapy (ERT)----1件: 19
224ERT----1件: 19
225EU-sourced Cerezyme1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
226FABRAZYME1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
227Fabrazyme (agalsidase beta)1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
228FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
229Fabrazyme 35 mg1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
230Fabrazyme 5 mg1件: Agalsidase beta1件: D03228 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
231FBX-101----1件: 19
232FLT190----1件: 19
233Fludarabina1件: Fludarabine2件: D01907 ,
D07966 		1件: RRM1 💬5件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬2件: 19, 65
234FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML1件: Fludarabine2件: D01907 ,
D07966 		1件: RRM1 💬5件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬1件: 19
235FLUDARABINA FOSFATO1件: Fludarabine2件: D01907 ,
D07966 		1件: RRM1 💬5件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬1件: 19
236Fluorescein1件: Fluorescein2件: D01261 ,
D02024 		--3件: 19, 22, 97
237GA-GCB1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
238GA-GCB (velaglucerase alfa)1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
239GABAPENTIN1件: Gabapentin1件: D00332 5件: CACNA2D1,
CACNA2D2,
CACNA2D3,
CACNA2D4,
SLC6A1 💬		9件: Adrenergic signaling in cardiomyocytes, Arrhythmogenic right ventricular cardiomyopathy, Cardiac muscle contraction, Dilated cardiomyopathy, GABAergic synapse, Hypertrophic cardiomyopathy, MAPK signaling pathway, Oxytocin signaling pathway, Synaptic vesicle cycle 💬5件: 6, 13, 19, 70, 298
240Galafold1件: Migalastat1件: D10359 --1件: 19
241GALSULFASE1件: Galsulfase1件: D06565 1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
242GC1111_0.5mg/kg----1件: 19
243GC1111_1.0mg/kg----1件: 19
244GC1119----1件: 19
245Gelatine1件: Gelatin2件: D00115 ,
D06892 		--1件: 19
246Gene Activated a-Galactosidase A----1件: 19
247Gene Activated Human Glucocerebrosidase----1件: 19
248Gene activated human glucocerebrosidase, velaglucerase alfa1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
249Gene-Activated Human Glucocerebrosidase----1件: 19
250Gene-Activated Human Glucocerebrosidase 200U/vial----1件: 19
251Gene-Activated Human Glucocerebrosidase 400U/vial----1件: 19
252Genetic HMI-203----1件: 19
253Genetic: GLA gene----1件: 19
254Genetic: LYS-GM101----1件: 19
255GENISTEIN1件: Genistein1件: D11680 --2件: 19, 299
256Genistein aglycone1件: Genistein1件: D11680 --1件: 19
257Genz-112638----1件: 19
258GENZ-6824521件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67
259Genz-682452-AA1件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
260Genz-682452-AU1件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67
261GLA----2件: 13, 19
262GLA genetic sequencing.----1件: 19
263GLUCOCEREBROSIDASE----1件: 19
264GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO----1件: 19
265GNR-055----1件: 19
266GR181413----1件: 19
267GR181413A----1件: 19
268GR181413A/AT1001 capsule----1件: 19
269GR181413A/AT1001 solution----1件: 19
270Growth hormone1件: Somatotropin1件: D02691 1件: GHR 💬5件: Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬13件: 2, 13, 19, 46, 78, 96, 107, 187, 191, 193, 195, 265, 299
271GSK2788723----1件: 19
272GZ/SAR402671----2件: 19, 67
273GZ385660----1件: 19
274GZ402665----1件: 19
275GZ402671----2件: 19, 67
276GZ402671 / SAR402671----1件: 19
277Hematopoetic Stem Cell Transplantation----5件: 19, 65, 120, 160, 326
278Hematopoietic stem cell infusion----2件: 19, 20
279Hematopoietic Stem Cell Transplantation----13件: 11, 13, 14, 19, 20, 35, 46, 49, 51, 65, 96, 234, 285
280Heparán N sulfatasa humana recombinante----1件: 19
281Heparán N sulfatasa humana recombinante (rhHNS)----1件: 19
282HGT-1110----1件: 19
283HGT-1111----1件: 19
284HGT-1410----1件: 19
285HGT-2310----1件: 19
286HIDS DONOR----1件: 19
287HuCNS-SC----1件: 19
288Human Glucocerebrosidase (prGCD)1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
289Human Placental Derived Stem Cell----4件: 19, 20, 60, 284
290Hunterase1件: Idursulfase beta1件: D11025 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
291Hydroxypropyl-beta-cyclodextrin1件: Betadex1件: D02401 --1件: 19
292Hydroxypropyl-ß-cyclodextrin----1件: 19
293HYDROXYPROPYLBETADEX----1件: 19
294Hydroxyurea1件: Hydroxyurea1件: D00341 1件: RRM2 💬6件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism, p53 signaling pathway 💬8件: 3, 13, 19, 20, 65, 85, 86, 284
295I2S----1件: 19
296I2S-IT----1件: 19
297IB1001----2件: 19, 65
298Iduronate 2-sulfatase1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
299Iduronate-2-sulfatase enzyme replacement therapy----1件: 19
300IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR----1件: 19
301IDURSULFASA1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
302IDURSULFASE1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
303Idursulfase (I2S)1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
304Idursulfase (I2S)-IT1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
305Idursulfase beta2件: Idursulfase,
Idursulfase beta		2件: D04499 ,
D11025 		1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
306Idursulfase IT (1 mg)1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
307Idursulfase IT (10 mg)1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
308Idursulfase IT (30 mg)1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
309Idursulfase(12S)-IT1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
310Idursulfase(I2S)-IT1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
311Idursulfase-IT1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
312IGF2----1件: 19
313IMD----5件: 19, 20, 125, 234, 326
314IMD Preparative Regimen----5件: 19, 20, 125, 234, 326
315IMIGLUCERASA1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
316IMIGLUCERASE1件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
317Imiglucerase GZ4378431件: Imiglucerase1件: D03020 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
318Imiglucerasi----1件: 19
319Imiglucérase (drug) pharmacokinetics----1件: 19
320Inactive Reminder Capsules----1件: 19
321Inactive Reminder Product----1件: 19
322INN Not available----2件: 19, 46
323INNOZIDE----1件: 19
324Intrathecal recombinant human alpha iduronidase----1件: 19
325Intraventricular Access Device----1件: 19
326Iohexol1件: Iohexol1件: D01817 --2件: 19, 67
327IRT Laronidase1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
328Isofagamine tartrate----1件: 19
329ISU302----1件: 19
330IV migalastat HCl1件: Migalastat1件: D10359 --1件: 19
331IVA 336----1件: 19
332JR-141----1件: 19
333JR-141 or Idursulfase1件: Idursulfase1件: D04499 1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
334JR-171----1件: 19
335KANUMA1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
336LAMAZYM1件: Velmanase alfa1件: D11024 1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19
337LARONIDASE1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬2件: 19, 256
338Laronidase ERT1件: Laronidase1件: D04670 1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
339Ledipasvir1件: Ledipasvir1件: D10442 --1件: 19
340Ledipasvir/Sofosbuvir2件: Ledipasvir,
Sofosbuvir		2件: D10366 ,
D10442 		--1件: 19
341Lenograstim1件: Lenograstim1件: D03247 1件: CSF3R 💬5件: Cytokine-cytokine receptor interaction, Hematopoietic cell lineage, JAK-STAT signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer 💬7件: 2, 13, 16, 18, 19, 57, 65
342Lentivirus Alpha-gal A transduced stem cells----1件: 19
343Lentivirus-mediated delivery of ARSA to the CNS.----1件: 19
344Leucine1件: Leucine1件: D00030 --11件: 5, 6, 7, 19, 65, 96, 97, 127, 283, 284, 299
345Leucovorin1件: Leucovorin3件: D01211 ,
D07986 ,
D07987 		--2件: 19, 46
346Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA)----1件: 19
347Lisinopril1件: Lisinopril2件: D00362 ,
D08131 		1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬7件: 19, 46, 49, 66, 67, 113, 222
348LITHIUM CARBONATE2件: Carbonate ion,
Lithium carbonate		1件: D00801 --6件: 2, 13, 17, 18, 19, 127
349Lopace----1件: 19
350Losartan1件: Losartan2件: D00357 ,
D08146 		1件: AGTR1 💬16件: AGE-RAGE signaling pathway in diabetic complications, Adrenergic signaling in cardiomyocytes, Aldosterone synthesis and secretion, Apelin signaling pathway, Calcium signaling pathway, Coronavirus disease - COVID-19, Cortisol synthesis and secretion, Cushing syndrome, Diabetic cardiomyopathy, Neuroactive ligand-receptor interaction, Pathways in cancer, Phospholipase D signaling pathway, Renin secretion, Renin-angiotensin system, Vascular smooth muscle contraction, cGMP-PKG signaling pathway 💬18件: 17, 19, 36, 46, 58, 66, 67, 85, 96, 97, 98, 113, 164, 167, 179, 215, 222, 299
351Losartan and other angiotensin receptor blockers2件: Angiotensin II,
Losartan		4件: D00150 ,
D00357 ,
D02014 ,
D08146 		2件: AGTR1,
AGTR2 💬		16件: AGE-RAGE signaling pathway in diabetic complications, Adrenergic signaling in cardiomyocytes, Aldosterone synthesis and secretion, Apelin signaling pathway, Calcium signaling pathway, Coronavirus disease - COVID-19, Cortisol synthesis and secretion, Cushing syndrome, Diabetic cardiomyopathy, Neuroactive ligand-receptor interaction, Pathways in cancer, Phospholipase D signaling pathway, Renin secretion, Renin-angiotensin system, Vascular smooth muscle contraction, cGMP-PKG signaling pathway 💬1件: 19
352Lucerastat1件: Lucerastat1件: D11439 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
353Lumbar Puncture----6件: 6, 13, 19, 96, 97, 127
354LYS----1件: 19
355LYS-GM101----1件: 19
356LYS-SAF302----1件: 19
357Lysodase----1件: 19
358Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)1件: Cholesterol1件: D00040 --1件: 19
359Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)1件: Cholesterol1件: D00040 --1件: 19
360MabThera1件: Rituximab1件: D02994 1件: MS4A1 💬1件: Hematopoietic cell lineage 💬22件: 11, 13, 14, 19, 35, 43, 44, 46, 49, 50, 51, 52, 53, 61, 63, 64, 65, 83, 93, 162, 222, 271
361MABTHERA - 2 FIALE 100 MG 10 ML1件: Rituximab1件: D02994 1件: MS4A1 💬1件: Hematopoietic cell lineage 💬2件: 19, 65
362Magnetic Resonance Imaging----7件: 6, 13, 19, 22, 34, 205, 299
363Mechanical stimulation with Von Frey filaments----1件: 19
364Medication to suppress the immune system----1件: 19
365Melphalan1件: Melphalan1件: D00369 --15件: 11, 13, 14, 19, 20, 28, 60, 62, 65, 96, 164, 234, 284, 285, 299
366Mepsevii1件: Vestronidase alfa1件: D11004 1件: GUSB 💬8件: Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬1件: 19
367Mercaptamina1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
368Mercaptamine1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
369MERCAPTAMINE BITARTRATE1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
370MERCAPTAMINE HYDROCHLORIDE1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
371Mercaptamine, mercamine, 2-aminoethanethiol1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
372Metazym----1件: 19
373Methotrexate1件: Methotrexate2件: D00142 ,
D02115 		2件: DHFR,
DHFR2 💬		5件: Antifolate resistance, Biosynthesis of cofactors, Folate biosynthesis, Metabolic pathways, One carbon pool by folate 💬38件: 11, 13, 19, 34, 35, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 53, 55, 56, 60, 62, 65, 84, 93, 96, 97, 107, 162, 164, 172, 256, 270, 271, 283, 284, 285, 286, 326, 331
374Methylprednisolone1件: Methylprednisolone6件: D00407 ,
D00751 ,
D00979 ,
D05000 ,
D05001 ,
D05002 		1件: NR3C1 💬1件: Neuroactive ligand-receptor interaction 💬48件: 2, 6, 11, 13, 14, 18, 19, 25, 26, 35, 38, 39, 40, 41, 42, 43, 45, 46, 49, 50, 51, 60, 62, 63, 64, 65, 66, 83, 84, 86, 93, 95, 96, 97, 127, 145, 162, 164, 211, 220, 222, 224, 271, 283, 284, 285, 296, 300
375MIGALASTAT1件: Migalastat1件: D10359 --1件: 19
376Migalastat HCl1件: Migalastat1件: D10359 --1件: 19
377Migalastat HCl 150 mg1件: Migalastat1件: D10359 --1件: 19
378MIGALASTAT HYDROCHLORIDE1件: Migalastat1件: D10359 --1件: 19
379Migalastat hydrocloride1件: Migalastat1件: D10359 --1件: 19
380MIGLUSTAT1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 19, 256, 299
381MIGLUSTAT (ZAVESCA)1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
382MONITOR1件: Methamidophos---20件: 2, 11, 13, 19, 28, 46, 49, 58, 65, 70, 81, 85, 86, 98, 113, 118, 129, 298, 299, 300
383Moss-aGal (recombinant human alpha-galactosidase A produced in moss)1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
384Mozobil1件: Plerixafor1件: D08971 1件: CXCR4 💬12件: Axon guidance, Calcium signaling pathway, Chemokine signaling pathway, Cytokine-cytokine receptor interaction, Endocytosis, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Intestinal immune network for IgA production, Leukocyte transendothelial migration, Pathways in cancer, Regulation of actin cytoskeleton, Viral protein interaction with cytokine and cytokine receptor 💬3件: 19, 65, 285
385MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F1件: Plerixafor1件: D08971 1件: CXCR4 💬12件: Axon guidance, Calcium signaling pathway, Chemokine signaling pathway, Cytokine-cytokine receptor interaction, Endocytosis, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Intestinal immune network for IgA production, Leukocyte transendothelial migration, Pathways in cancer, Regulation of actin cytoskeleton, Viral protein interaction with cytokine and cytokine receptor 💬1件: 19
386MPS----1件: 19
387MRI with contrast agent injection----1件: 19
388MRI without contrast agent injection----1件: 19
389Mycophenolate1件: Mycophenolic acid1件: D05096 2件: IMPDH1,
IMPDH2 💬		3件: Drug metabolism - other enzymes, Metabolic pathways, Purine metabolism 💬36件: 2, 11, 13, 19, 20, 28, 35, 36, 42, 43, 44, 45, 49, 51, 53, 60, 61, 62, 65, 66, 84, 85, 93, 95, 96, 162, 164, 222, 224, 226, 228, 234, 283, 284, 285, 300
390Mycophenolate mofetil2件: Mycophenolate mofetil,
Mycophenolic acid		4件: D00752 ,
D05094 ,
D05095 ,
D05096 		2件: IMPDH1,
IMPDH2 💬		3件: Drug metabolism - other enzymes, Metabolic pathways, Purine metabolism 💬34件: 2, 11, 13, 19, 20, 28, 35, 36, 42, 43, 44, 45, 49, 51, 53, 60, 61, 62, 65, 66, 85, 93, 95, 96, 162, 164, 222, 224, 226, 234, 283, 284, 285, 300
391Mycophenylate mofetil----2件: 19, 20
392MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML----2件: 19, 65
393N acetyl cysteine1件: Cysteine2件: D00026 ,
D02326 		--2件: 6, 19
394N Acetyl L Leucine1件: Leucine1件: D00030 --1件: 19
395N-acetyl Cysteine1件: Cysteine2件: D00026 ,
D02326 		--7件: 13, 19, 34, 85, 88, 296, 310
396N-Acetyl-L Leucine1件: Leucine1件: D00030 --1件: 19
397N-Acetyl-L-Leucine1件: Leucine1件: D00030 --2件: 19, 65
398N-Acetylcysteine1件: Acetylcysteine1件: D00221 --11件: 6, 13, 19, 20, 49, 58, 85, 111, 164, 299, 337
399N-acetylgalactosamine 4-sulfatase----1件: 19
400Naglazyme1件: Galsulfase1件: D06565 1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
401Naglazyme (galsulfase)1件: Galsulfase1件: D06565 1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
402Naglazyme®1件: Galsulfase1件: D06565 1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
403Non applicable----2件: 10, 19
404Odiparcil1件: Odiparcil---1件: 19
405OGT 918----2件: 19, 299
406OGT918----1件: 19
407OGT923----1件: 19
408OLENASUFLIGENE RELDUPARVOVEC1件: Olenasufligene relduparvovec---1件: 19
409Olipudase alfa1件: Olipudase alfa1件: D10820 1件: SMPD1 💬5件: Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬1件: 19
410Olipudase alfa (rhASM)1件: Olipudase alfa1件: D10820 1件: SMPD1 💬5件: Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬1件: 19
411OMNIPAQUE1件: Iohexol1件: D01817 --2件: 19, 222
412Ophthalmology exam----1件: 19
413Oral migalastat HCl1件: Migalastat1件: D10359 --1件: 19
414OSOME----1件: 19
415Osteopetrosis Haploidentical Only Preparative Regimen----5件: 19, 20, 125, 234, 326
416Osteopetrosis Only Preparative Regimen----5件: 19, 20, 125, 234, 326
417Other: Brief Pain Inventory questionnaire----1件: 19
418Other: Challenge agent: capsaicin1件: Capsaicin1件: D00250 1件: TRPV1 💬2件: Inflammatory mediator regulation of TRP channels, Neuroactive ligand-receptor interaction 💬1件: 19
419Other: Control----9件: 6, 19, 67, 70, 75, 85, 97, 162, 193
420Other: Cysteamine bitartrate1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
421Other: Dried blood spot test for MPS----1件: 19
422Other: Electroencephalogram (EEG) awake andextended overnight----1件: 19
423Other: Fluorescein angiography1件: Fluorescein2件: D01261 ,
D02024 		--1件: 19
424Other: General and Neurological examination----1件: 19
425Other: Ketogenic Diet----2件: 19, 67
426Other: MPS I----1件: 19
427Other: Neurology exam----1件: 19
428Other: No IT treatment----1件: 19
429Other: Palliative Care----5件: 19, 65, 120, 160, 326
430Other: Parts A/B: Sham Control----1件: 19
431Other: Potable water1件: Water1件: D00001 --1件: 19
432Other: Quality of Life questionnaire----1件: 19
433Other: Vital signs----1件: 19
434OTL-200----1件: 19
435OTL-200 Dispersion for Infusion----1件: 19
436Oxygen1件: Oxygen1件: D00003 --20件: 6, 13, 19, 46, 49, 70, 84, 85, 86, 88, 90, 96, 97, 168, 193, 226, 271, 294, 299, 330
437Oxygen flow at the optic nerve head measurement1件: Oxygen1件: D00003 --1件: 19
438Pabinafusp alfa1件: Pabinafusp alfa1件: D11812 2件: IDS,
TFRC 💬		8件: Endocytosis, Ferroptosis, Glycosaminoglycan degradation, HIF-1 signaling pathway, Hematopoietic cell lineage, Lysosome, Metabolic pathways, Phagosome 💬1件: 19
439Paricalcitol1件: Paricalcitol1件: D00930 1件: VDR 💬5件: Chemical carcinogenesis - receptor activation, Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis 💬4件: 19, 66, 218, 238
440Parts A/B: Adrabetadex1件: Adrabetadex---1件: 19
441PBGM01----1件: 19
442PBKR03----1件: 19
443PEGUNIGALSIDASE ALFA1件: Pegunigalsidase alfa1件: D11685 --1件: 19
444Pegunigalsidase alpha----1件: 19
445Pending----6件: 13, 19, 96, 97, 113, 302
446Pentosan Polysulfat SP 54® injection solution----1件: 19
447Pentosan polysulfate1件: Pentosan polysulfate1件: D05428 --3件: 19, 26, 226
448PENTOSAN POLYSULFATE SODIUM1件: Pentosan polysulfate1件: D05428 --1件: 19
449Pentosan Polysulphate SP54----1件: 19
450Pentosan Polysulphate SP54®----1件: 19
451Perdix----1件: 19
452Plant cell expressed recombinant glucocerebrosidase (prGCD)1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
453Plant Cell Recombinant Glucocerebrosidase----1件: 19
454Plasma Lyso-GB3----1件: 19
455PLX-200----1件: 19
456PR001----2件: 6, 19
457Prednisone1件: Prednisone1件: D00473 1件: NR3C1 💬1件: Neuroactive ligand-receptor interaction 💬48件: 2, 6, 11, 13, 14, 19, 26, 35, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 53, 61, 63, 64, 65, 66, 79, 81, 84, 85, 86, 95, 96, 97, 113, 127, 162, 164, 205, 222, 224, 228, 251, 283, 284, 285, 288, 299, 300, 331
458Prevenar13----2件: 19, 265
459PrGCD1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
460PROCYSBI1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
461PRX-102----1件: 19
462PRX-102 (pegunigalsidase alfa)1件: Pegunigalsidase alfa1件: D11685 --1件: 19
463PRX-112----1件: 19
464PRX102----1件: 19
465PTC124----4件: 19, 113, 246, 299
466PYRIMETHAMINE1件: Pyrimethamine1件: D00488 --4件: 2, 4, 19, 65
467Quantitative chemical shift imaging (QCSI)----1件: 19
468RAAV 2/6 hIDS DONOR----1件: 19
469RAAV2/5-hNAGLU----1件: 19
470RAAV2/6 Left ZFN Vector----1件: 19
471RAAV2/6 Right ZFN Vector----1件: 19
472RAAV9.CMV.hNAGLU----1件: 19
473Radiation: Total Body Irradiation with Marrow Boosting----2件: 19, 234
474Rebisufligene etisparvovec----1件: 19
475Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A----1件: 19
476Recombinant alpha-galactosidase A1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
477RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)----1件: 19
478Recombinant human alpha-galactosidase----1件: 19
479Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL----1件: 19
480Recombinant human alpha-mannosidase----1件: 19
481Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)----1件: 19
482RECOMBINANT HUMAN ARYLSULFATASE A----1件: 19
483Recombinant Human Arylsulfatase A (rhASA)----1件: 19
484Recombinant Human Arylsulfatase A (rhASA,----1件: 19
485Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase----1件: 19
486RECOMBINANT HUMAN BETA GLUCURONIDASE----1件: 19
487Recombinant human beta-glucuronidase1件: Vestronidase alfa1件: D11004 1件: GUSB 💬8件: Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬1件: 19
488Recombinant human beta-glucuronidase (rhGUS)1件: Vestronidase alfa1件: D11004 1件: GUSB 💬8件: Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬1件: 19
489Recombinant human betaglucuronidase----1件: 19
490Recombinant human derived macrophage-targeted beta-Glucocerebrosidase----1件: 19
491Recombinant human derived macrophage-targeted ß-Glucocerebrosidase----1件: 19
492Recombinant human glucerebrosidase----1件: 19
493Recombinant human glucocerebrosidase----1件: 19
494Recombinant Human Glucocerebrosidase (prGCD)1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
495Recombinant human heparan N-sulfatase----1件: 19
496Recombinant human heparan N-sulfatase (rhHNS)----1件: 19
497Recombinant human heparan N-sulfatase [rhHNS]----1件: 19
498Recombinant human heparan Nsulfatase (rhHNS)----1件: 19
499Recombinant human lysosomal acid lipase----1件: 19
500Recombinant human lysosomal acid lipase (rhLAL)----1件: 19
501Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)1件: Cholesterol1件: D00040 --1件: 19
502Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA)1件: Cholesterol1件: D00040 --1件: 19
503Recombinant human lysosomal alpha-mannosidase----1件: 19
504Recombinant human N-acetylgalactosamine 4-sulfatase----1件: 19
505Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110----1件: 19
506Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance----1件: 19
507Recombinant human N-acetylgalactosamine-6-sulfatase1件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
508Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)1件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
509Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 1101件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
510Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance1件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
511Recombinant human Nacetylgalactosamine-----1件: 19
512Recombinant human tripeptidyl peptidase-1----1件: 19
513RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)----1件: 19
514Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene----1件: 19
515Reduced Intensity Conditioning----2件: 19, 65
516Reminder capsules----1件: 19
517Reminder Product----1件: 19
518REPLAGAL1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
519Replagal (Agalsidase Alfa)1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
520Replagal agalsidase alfa1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
521REPLAGAL ®1件: Agalsidase alfa1件: D02784 1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19
522Rh Growth Hormone1件: Somatotropin1件: D02691 1件: GHR 💬5件: Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬1件: 19
523RhARSA----1件: 19
524RhASA----1件: 19
525RhASA - Dose Level 1----1件: 19
526RhASA - Dose Level 2----1件: 19
527RhASA - Dose Level 3----1件: 19
528RhASM----1件: 19
529RhGALNS1件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
530RHGUS----1件: 19
531RhHNS-10 mg----1件: 19
532RhHNS-45 mg----1件: 19
533RhHNS-90 mg----1件: 19
534RhIDU (recombinant human-Alpha-L-Iduronidase)----1件: 19
535RhLAMAN1件: Velmanase alfa1件: D11024 1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19
536RhNAGLU----1件: 19
537RhNAGLU radiolabelled----1件: 19
538RHNAGLU-IGF2----1件: 19
539Rimiducid1件: Rimiducid1件: D11195 1件: CASP9 💬35件: Alzheimer disease, Amyotrophic lateral sclerosis, Apoptosis, Apoptosis - multiple species, Colorectal cancer, Endometrial cancer, Epstein-Barr virus infection, Hepatitis B, Hepatitis C, Herpes simplex virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Huntington disease, Influenza A, Kaposi sarcoma-associated herpesvirus infection, Legionellosis, Lipid and atherosclerosis, Measles, Non-small cell lung cancer, PI3K-Akt signaling pathway, Pancreatic cancer, Parkinson disease, Pathogenic Escherichia coli infection, Pathways in cancer, Pathways of neurodegeneration - multiple diseases, Platinum drug resistance, Prion disease, Prostate cancer, Small cell lung cancer, Thyroid hormone signaling pathway, Toxoplasmosis, Tuberculosis, VEGF signaling pathway, Viral myocarditis, p53 signaling pathway 💬6件: 19, 60, 65, 284, 285, 326
540Rituximab1件: Rituximab1件: D02994 1件: MS4A1 💬1件: Hematopoietic cell lineage 💬49件: 6, 11, 13, 14, 17, 19, 35, 36, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 53, 55, 56, 60, 61, 63, 64, 65, 66, 83, 84, 85, 86, 93, 94, 96, 97, 127, 162, 222, 229, 256, 271, 274, 283, 284, 285, 288, 296, 300, 331
541Rivogenlecleucel----6件: 19, 60, 65, 284, 285, 326
542RP 103----2件: 8, 19
543RP103----2件: 8, 19
544RVX000222----1件: 19
545SAF-301----1件: 19
546Saline Solution for Injection----1件: 19
547SAR402671, GZ402671 o GZ/SAR402671----2件: 19, 67
548SAR402671, GZ402671 or GZ / SAR402671----1件: 19
549SAR402671, GZ402671 or GZ/SAR402671----3件: 6, 19, 67
550SB-318----1件: 19
551SB-47171----1件: 19
552SB-47898----1件: 19
553SB-913----1件: 19
554SB-A6P-HRL Donor Vector----1件: 19
555SB-FIX----1件: 19
556SB-IDUA----1件: 19
557SBC-1021件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
558SBC-102, recombinant human lysosomal acid lipase1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
559SBC-103----1件: 19
560ScAAV9.CB.CLN6----1件: 19
561ScAAV9.U1A.hSGSH----1件: 19
562ScAAV9.U1A.SGSH----1件: 19
563Sebelipasa Alfa1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
564Sebelipase Alfa1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
565Sebelipase alfa (SBC-102)1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
566Sebelipase alfa 0.35 mg/kg1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
567Sebelipase alfa 1 mg/kg1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
568Sebelipase alfa 3 mg/kg1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
569SHP611----1件: 19
570SHP675----1件: 19
571Sirolimus1件: Sirolimus1件: D00753 1件: MTOR 💬51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, EGFR tyrosine kinase inhibitor resistance, Endocrine resistance, 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 💬36件: 6, 13, 15, 17, 19, 34, 35, 36, 46, 49, 51, 60, 61, 62, 63, 65, 66, 67, 84, 85, 89, 98, 127, 137, 157, 158, 192, 222, 277, 278, 279, 280, 281, 283, 285, 331
572Skeletal survey----1件: 19
573Skin biopsy----2件: 19, 84
574Slit Lamp assessment and intra-ocular pressure measurement----1件: 19
575SOBI003----1件: 19
576Sofosbuvir1件: Sofosbuvir1件: D10366 --1件: 19
577Somatropin1件: Somatotropin1件: D02691 1件: GHR 💬5件: Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬19件: 2, 3, 19, 46, 65, 74, 75, 78, 96, 107, 113, 187, 193, 195, 238, 265, 274, 276, 299
578Somatropin (DNA origin)1件: Somatotropin1件: D02691 1件: GHR 💬5件: Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬1件: 19
579Speech and modified barium swallow study1件: Barium---1件: 19
580Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride1件: Cysteamine2件: D03634 ,
D03635 		--1件: 19
581ST-920----1件: 19
582Stem Cell Transplant----6件: 19, 20, 60, 65, 164, 284
583Stem Cell Transplantation----13件: 13, 19, 20, 46, 51, 60, 62, 65, 90, 125, 164, 234, 326
584SUB195712----1件: 19
585Surgery to implant human CNS stem cells (HuCNS-SC)----1件: 19
586TALIGLUCERASE ALFA1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
587Taliglucerase alfa - Recombinant human glucocerebrosidase1件: Taliglucerase alfa1件: D09675 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
588TBD----4件: 19, 86, 276, 299
589Temperature sensitivity measurement with Advanced Thermal Stimulation----1件: 19
590Thiotepa1件: Thiotepa1件: D00583 --12件: 13, 19, 20, 28, 51, 60, 65, 85, 96, 164, 284, 285
591Thiotepa--escalated dose1件: Thiotepa1件: D00583 --4件: 19, 20, 65, 284
592Thiotepa--single daily dose1件: Thiotepa1件: D00583 --4件: 19, 20, 65, 284
593Thyroxine1件: Levothyroxine2件: D01010 ,
D08125 		2件: THRA,
THRB 💬		2件: Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬1件: 19
594To be added after EV code is available----1件: 19
595Total body Irradiation----6件: 19, 20, 51, 60, 65, 285
596Trappsol Cyclo----1件: 19
597Trappsol® Cyclo™----1件: 19
598TREOSULFAN1件: Treosulfan1件: D07253 --6件: 13, 19, 62, 65, 283, 286
599Triapin----1件: 19
600Triapin& triapin Mite----1件: 19
601Tropicamide1件: Tropicamide1件: D00397 5件: CHRM1,
CHRM2,
CHRM3,
CHRM4,
CHRM5 💬		13件: Alzheimer disease, Calcium signaling pathway, Cholinergic synapse, Gastric acid secretion, Insulin secretion, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway, Pancreatic secretion, Pathways of neurodegeneration - multiple diseases, Regulation of actin cytoskeleton, Salivary secretion, Taste transduction, cAMP signaling pathway 💬2件: 6, 19
602TSHA-101----1件: 19
603Unrelated Umbilical Cord Blood Transplant----1件: 19
604UPLYSO----1件: 19
605Urine collection----1件: 19
606USAN: sebelipasa alfa1件: Sebelipase alfa1件: D10377 1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19
607USAN: sebelipase alpha----1件: 19
608UX003----1件: 19
609Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana----1件: 19
610VELAGLUCERASE ALFA1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
611Velaglucerase alfa (VPRIV)1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
612Velaglucerasi alfa----1件: 19
613VELMANASE ALFA1件: Velmanase alfa1件: D11024 1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19
614Velmanase Alfa (e.g. Lamazym)1件: Velmanase alfa1件: D11024 1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19
615Venglustat1件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67
616Venglustat (GZ402671)1件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
617Venglustat GZ4026711件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬2件: 19, 67
618Venglustat malate1件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67
619Venglustat malato1件: Venglustat1件: D11633 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬2件: 19, 67
620Vestronidase alfa1件: Vestronidase alfa1件: D11004 1件: GUSB 💬8件: Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬1件: 19
621Vimizim® (elosulfase alfa)1件: Elosulfase alfa1件: D10333 1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19
622Visual field testing----1件: 19
623Vitamin D1件: Vitamin D---24件: 3, 13, 15, 19, 41, 43, 44, 46, 49, 50, 68, 93, 95, 96, 97, 98, 179, 222, 228, 235, 256, 274, 298, 299
624Vorinostat1件: Vorinostat1件: D06320 11件: HDAC1,
HDAC10,
HDAC11,
HDAC2,
HDAC3,
HDAC4,
HDAC5,
HDAC6,
HDAC7,
HDAC8,
HDAC9 💬		17件: Alcoholism, Amphetamine addiction, Amyotrophic lateral sclerosis, Apelin signaling pathway, Cell cycle, Chronic myeloid leukemia, Epstein-Barr virus infection, Human papillomavirus infection, Huntington disease, Longevity regulating pathway - multiple species, MicroRNAs in cancer, Neutrophil extracellular trap formation, Notch signaling pathway, Pathways in cancer, Thyroid hormone signaling pathway, Transcriptional misregulation in cancer, Viral carcinogenesis 💬3件: 19, 75, 96
625VPRIV1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
626VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
627VPRIV 400 Units powder for solution of infusion1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
628VPRIV ®,1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
629VPRIV®1件: Velaglucerase alfa1件: D09029 1件: GBA 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19
630VTS-270----1件: 19
631Warfarin1件: Warfarin3件: D00564 ,
D01280 ,
D08682 		2件: NQO1,
VKORC1 💬		7件: Biosynthesis of cofactors, Chemical carcinogenesis - reactive oxygen species, Fluid shear stress and atherosclerosis, Hepatocellular carcinoma, Metabolic pathways, Pathways in cancer, Ubiquinone and other terpenoid-quinone biosynthesis 💬13件: 6, 8, 13, 19, 46, 49, 66, 85, 86, 88, 91, 96, 97
632Water1件: Water1件: D00001 --23件: 2, 5, 6, 13, 17, 19, 22, 35, 46, 51, 53, 56, 57, 63, 65, 67, 78, 84, 96, 97, 226, 278, 299
633Whole body Protein turnover----4件: 19, 46, 96, 299
634ZAVESCA1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 19, 256, 299
635Zavesca (Miglustat)1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
636ZAVESCA 84CPS 100MG1件: Miglustat1件: D05032 1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19
637Zestoretic 10,201件: Lisinopril2件: D00362 ,
D08131 		1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬1件: 19
638ZESTORIC----1件: 19
639Zestril1件: Lisinopril2件: D00362 ,
D08131 		1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬3件: 19, 67, 222