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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: Migalastat 💬--1件: 19 💬
41-deoxygalactonojirimycin hydrochloride1件: Migalastat1件: Migalastat 💬--1件: 19 💬
512S-IT----1件: 19 💬
615-0 H20----2件: 6, 19 💬
72(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID----2件: 19, 65 💬
82-aminoethanethiol1件: Cysteamine1件: Cysteamine 💬--2件: 19, 299 💬
92-HYDROXYPROPYL-BETA-CYCLODEXTRIN2件: Betadex,
Hydroxypropyl betadex		1件: Betadex 💬--1件: 19 💬
1026 common Fabry mutation types in Taiwan----1件: 19 💬
114D-310----1件: 19 💬
12500 0500 0----1件: 19 💬
1389 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)----1件: 19 💬
1489Zr-SBC-103----1件: 19 💬
15[14C] AT1001----1件: 19 💬
16A (migalastat)1件: Migalastat1件: Migalastat 💬--1件: 19 💬
17A16AB04----1件: 19 💬
18A16AX06----1件: 19 💬
19AAV2/8.TBG.hARSB----1件: 19 💬
20AAV2CUhCLN2 (3x10^12 particle units)----1件: 19 💬
21AAV9-CAG-coh-SGSH----1件: 19 💬
22AAV9-GLB1----1件: 19 💬
23AAVrh.10-hMPSIIIA----1件: 19 💬
24AAVrh.10cuARSA----1件: 19 💬
25AAVrh.10CUCLN2----1件: 19 💬
26AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies----1件: 19 💬
27AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies----1件: 19 💬
28Abatacept1件: Abatacept1件: Abatacept 💬2件: CD80,
CD86 💬		13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, Virion - Adenovirus 💬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 💬
29Abcertin----1件: 19 💬
30ABO-101----1件: 19 💬
31ABO-102----1件: 19 💬
32Accupro----1件: 19 💬
33Accuretic1件: Quinapril1件: Quinapril 💬1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬1件: 19 💬
34Acetyl-l-leucine2件: L-Acetylleucine,
Leucine		1件: Leucine 💬--2件: 19, 65 💬
35Acetylcysteine1件: Acetylcysteine1件: Acetylcysteine 💬--21件: 6, 13, 19, 20, 21, 49, 51, 53, 58, 63, 85, 90, 94, 111, 164, 193, 233, 296, 298, 299, 337 💬
36ACT-434964----1件: 19 💬
37Adalimumab1件: Adalimumab1件: Adalimumab 💬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 💬17件: 19, 37, 40, 41, 46, 56, 84, 96, 97, 107, 160, 164, 222, 226, 269, 270, 271 💬
38ADENO-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: Levothyroxine1件: Levothyroxine 💬2件: THRA,
THRB 💬		2件: Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬1件: 19 💬
39Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene----1件: 19 💬
40Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-betagal)----1件: 19 💬
41Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal)----1件: 19 💬
42Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
43Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
44Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector----1件: 19 💬
45Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector----1件: 19 💬
46Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector----1件: 19 💬
47Adeno-associated virus serotype hu68 containing the human GALC gene----1件: 19 💬
48Adeno-associated virus serotype rh.10 expressing beta-galactosidase1件: Tilactase1件: Lactase 💬--1件: 19 💬
49ADMINISTRATION----1件: 19 💬
50Adrabetadex1件: Adrabetadex---1件: 19 💬
51Afegostat1件: Afegostat1件: Afegostat 💬--1件: 19 💬
52Afegostat tartrate1件: Afegostat1件: Afegostat 💬--1件: 19 💬
53Agalsidase----1件: 19 💬
54AGALSIDASE ALFA1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
55Agalsidase Alpha1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
56AGALSIDASE BETA1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
57Agalsidase beta (GZ419828)1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
58Agalsidase beta (recombinant form)1件: Agalsidase beta1件: Agalsidase beta 💬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 from Biosidus 1 mg/kg1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
60AGALSIDASI ALFA----1件: 19 💬
61AGALSIDASI BETA----1件: 19 💬
62AGT-181----1件: 19 💬
63AGT-181 (HIRMAb-IDUA)----1件: 19 💬
64AGT-182----1件: 19 💬
65Aldurazyme1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
66Aldurazyme (laronidase)1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬2件: 19, 256 💬
67Aldurazyme (Recombinant Human Alpha-L-Iduronidase)1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
68ALDURAZYME*INFUS 1FL 5ML 500U1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
69ALEMTUZUMAB1件: Alemtuzumab1件: Alemtuzumab 💬1件: CD52 💬-17件: 13, 15, 19, 20, 36, 43, 44, 46, 51, 60, 63, 65, 85, 96, 164, 283, 284 💬
70Alendronate1件: Alendronic acid1件: Alendronic acid 💬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 💬
71Alendronate sodium1件: Alendronic acid1件: Alendronic acid 💬1件: FDPS 💬4件: Human T-cell leukemia virus 1 infection, Influenza A, Metabolic pathways, Terpenoid backbone biosynthesis 💬2件: 19, 299 💬
72Alfa-manosidadas humana recombinante----1件: 19 💬
73ALISERIN - 25 MG GRANULATO EFFERVESCENTE 20 BUSTINE----1件: 19 💬
74Alpha-galactosidase A1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
75Ambroxol1件: Ambroxol1件: Ambroxol 💬--2件: 6, 19 💬
76Ambroxol Hydrochloride1件: Ambroxol1件: Ambroxol 💬--2件: 6, 19 💬
77AMBROXOL HYDROCHLORIDE PH. EUR.1件: Ambroxol1件: Ambroxol 💬--1件: 19 💬
78Angiotensin1件: Angiotensin II2件: Angiotensin II ,
Angiotensin II 💬		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: Arimoclomol 💬--3件: 2, 15, 19 💬
80Arimoclomol (hard gelatine capsule)2件: Arimoclomol,
Gelatin		3件: Gelatin ,
Donkey glue ,
Arimoclomol 💬		--1件: 19 💬
81Arimoclomol (HPMC capsule)1件: Arimoclomol1件: Arimoclomol 💬--1件: 19 💬
82Arimoclomol citrate1件: Arimoclomol1件: Arimoclomol 💬--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: Ataluren 💬--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 CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII----1件: 19 💬
92Autologous Plasmablasts----1件: 19 💬
93Autologous Plasmablasts (B cells)----1件: 19 💬
94AVR-RD-01----1件: 19 💬
95AVR-RD-02----1件: 19 💬
96AX----1件: 19 💬
97AX 250----1件: 19 💬
98AXO-AAV-GM2 High Dose----1件: 19 💬
99AXO-AAV-GM2 Low Dose----1件: 19 💬
100AXO-AAV-GM2 Middle Dose----1件: 19 💬
101AXO-AAV-GM2 Starting Dose----1件: 19 💬
102Azathioprine1件: Azathioprine1件: Azathioprine 💬--21件: 6, 11, 13, 19, 35, 42, 43, 44, 45, 46, 49, 63, 65, 66, 85, 93, 94, 95, 96, 97, 162 💬
103Azathioprine (AZA)1件: Azathioprine1件: Azathioprine 💬--3件: 13, 19, 97 💬
104B (migalastat)1件: Migalastat1件: Migalastat 💬--1件: 19 💬
105Beta-cyclodextrin1件: Betadex1件: Betadex 💬--1件: 19 💬
106Beta-Galactosidase1件: Tilactase1件: Lactase 💬--1件: 19 💬
107Beta-Mercaptoethylamine1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
108Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
109BHP001----1件: 19 💬
110Blood sampling for biological and genetic analysis----1件: 19 💬
111BM110----1件: 19 💬
112BMN 110----1件: 19 💬
113BMN 110 - Every Other Week----1件: 19 💬
114BMN 110 - Weekly----1件: 19 💬
115BMN 110 Every Other Week----1件: 19 💬
116BMN 110 Weekly----1件: 19 💬
117BMN 190----1件: 19 💬
118BMN 250----1件: 19 💬
119BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)----1件: 19 💬
120Brineura1件: Cerliponase alfa1件: Cerliponase alfa 💬1件: TPP1 💬1件: Lysosome 💬1件: 19 💬
121BUSILVEX1件: Busulfan1件: Busulfan 💬--4件: 19, 36, 60, 65 💬
122BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI1件: Busulfan1件: Busulfan 💬--2件: 19, 65 💬
123Busulfan1件: Busulfan1件: Busulfan 💬--13件: 19, 20, 28, 36, 49, 51, 60, 65, 164, 274, 284, 285, 326 💬
124Busulfan, Cyclophosphamide, Antithymocyte Globulin2件: Busulfan,
Cyclophosphamide		3件: Busulfan ,
Cyclophosphamide ,
Cyclophosphamide 💬		--2件: 19, 20 💬
125Busulfan, Cyclophosphamide, ATG2件: Busulfan,
Cyclophosphamide		3件: Busulfan ,
Cyclophosphamide ,
Cyclophosphamide 💬		--1件: 19 💬
126BUSULFANO1件: Busulfan1件: Busulfan 💬--1件: 19 💬
127C (migalastat)1件: Migalastat1件: Migalastat 💬--1件: 19 💬
128Calcium2件: Calcium,
Calcium carbonate		2件: Calcium carbonate ,
Calcium carbonate 💬		--27件: 6, 13, 19, 46, 49, 51, 53, 56, 68, 75, 79, 84, 93, 94, 95, 96, 97, 156, 214, 215, 222, 224, 235, 265, 274, 298, 299 💬
129Calcium carbonate3件: Calcium,
Calcium carbonate,
Carbonate ion		2件: Calcium carbonate ,
Calcium carbonate 💬		--8件: 13, 19, 46, 53, 75, 96, 235, 299 💬
130CALD HR-D (High-Risk, Regimen C)----5件: 19, 20, 125, 234, 326 💬
131CALD HR-D (High-Risk, Regimen D)----5件: 19, 20, 125, 234, 326 💬
132CALD SR-A (Standard-Risk, Regimen A)----5件: 19, 20, 125, 234, 326 💬
133CALD SR-B (Standard-Risk, Regimen B)----5件: 19, 20, 125, 234, 326 💬
134Campath1件: Alemtuzumab1件: Alemtuzumab 💬1件: CD52 💬-12件: 19, 20, 49, 51, 60, 62, 65, 164, 234, 283, 285, 326 💬
135Campath-1H1件: Alemtuzumab1件: Alemtuzumab 💬1件: CD52 💬-6件: 19, 20, 60, 234, 285, 326 💬
136Cannabis1件: Medical Cannabis---7件: 2, 6, 13, 19, 90, 96, 97 💬
137Cannabis sativa L., folium cum flore1件: Medical Cannabis---1件: 19 💬
138Carbonate1件: Carbonate ion---12件: 2, 13, 17, 18, 19, 46, 53, 75, 96, 127, 235, 299 💬
139Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi----1件: 19 💬
140Cerdelga1件: Eliglustat1件: Eliglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
141Ceredase1件: Alglucerase1件: Alglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
142CEREDASE™1件: Alglucerase1件: Alglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
143CEREZYME1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
144Cerezyme (imiglucerase for injection)1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
145Cerezyme 400 Units Powder for concentrate for solution for infusion1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
146Cerezyme®1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
147Cerezyme® / Imiglucerase1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
148Cerliponase Alfa1件: Cerliponase alfa1件: Cerliponase alfa 💬1件: TPP1 💬1件: Lysosome 💬1件: 19 💬
149CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE----1件: 19 💬
150CHF6657----1件: 19 💬
151CHOLECALCIFEROL1件: Cholecalciferol1件: Cholecalciferol 💬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 💬9件: 13, 19, 34, 46, 49, 96, 97, 298, 299 💬
152Cholesterol1件: Cholesterol1件: Cholesterol 💬--6件: 8, 19, 90, 160, 222, 310 💬
153Clofarabine1件: Clofarabine1件: Clofarabine 💬3件: POLA1,
POLA2,
RRM1 💬		7件: DNA replication, Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬5件: 19, 20, 60, 234, 326 💬
154Coversyl1件: Perindopril1件: Perindopril 💬1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬2件: 19, 167 💬
155Coversyl PLUS----1件: 19 💬
156CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE----1件: 19 💬
157CsA----8件: 19, 49, 50, 53, 60, 63, 222, 228 💬
158CTNS-RD-04 or CTNS-RD-04-LB (where the suffix -LB stands for LentiBOOST)----1件: 19 💬
159Cyclophosphamide1件: Cyclophosphamide2件: Cyclophosphamide ,
Cyclophosphamide 💬		--44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, 274, 283, 284, 285, 288, 326, 331 💬
160Cyclosporine1件: Cyclosporine1件: Cyclosporine 💬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 💬
161Cyclosporine A1件: Cyclosporine1件: Cyclosporine 💬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 💬
162Cyclosporine A (CsA)1件: Cyclosporine1件: Cyclosporine 💬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 💬
163Cystadane1件: Betaine1件: Betaine 💬--1件: 19 💬
164Cystadane anhydrous1件: Betaine1件: Betaine 💬--1件: 19 💬
165CYSTADROPS1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
166CYSTADROPS 0.55% eye drops, solution1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
167CYSTAGON1件: Cysteamine1件: Cysteamine 💬--3件: 8, 19, 299 💬
168Cystagon 150mg1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
169Cystagon 50mg1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
170Cystagon®1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
171Cystagon® (Cysteamine Bitartrate)1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
172Cysteamine1件: Cysteamine1件: Cysteamine 💬--4件: 8, 19, 21, 299 💬
173Cysteamine (mercaptamine) viscous solution1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
174Cysteamine Bitartrate1件: Cysteamine1件: Cysteamine 💬--4件: 8, 19, 21, 299 💬
175Cysteamine bitartrate (INN: mercaptamine bitartrate1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
176Cysteamine bitartrate (INN: mercaptamine bitartrate )1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
177Cysteamine bitartrate (INN: mercaptamine bitartrate)1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
178Cysteamine Bitartrate Delayed-release Capsules1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
179Cysteamine Bitartrate Delayed-release Capsules (RP103)1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
180CYSTEAMINE HYDROCHLORIDE1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
181Cysteine1件: Cysteine---10件: 13, 19, 34, 84, 85, 88, 90, 254, 296, 310 💬
182D (migalastat)1件: Migalastat1件: Migalastat 💬--1件: 19 💬
183Dapagliflozin1件: Dapagliflozin1件: Dapagliflozin 💬1件: SLC5A2 💬-7件: 19, 49, 66, 67, 86, 88, 222 💬
184Dapagliflozin 10mg Tab1件: Dapagliflozin1件: Dapagliflozin 💬1件: SLC5A2 💬-1件: 19 💬
185Diagnosis of Fabry disease----1件: 19 💬
186DIFENIDRAMINA CLORIDRATO----3件: 11, 19, 63 💬
187DNL310----1件: 19 💬
188DNL310 Drug Substance----1件: 19 💬
189Dried blood spot (DBS) sampling----1件: 19 💬
190DRX005B----1件: 19 💬
191DUOC-01----3件: 13, 19, 20 💬
192E (migalastat)1件: Migalastat1件: Migalastat 💬--1件: 19 💬
193Elaprase1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
194Elaprase 2mg/ml1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
195Elaprase for intravenous (IV) infusion1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
196Elaprase_0.5mg/kg----1件: 19 💬
197Elelyso1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
198Elelyso 60 units/kg1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
199Eliglustat1件: Eliglustat1件: Eliglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
200ELIGLUSTAT TARTRATE1件: Eliglustat1件: Eliglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
201Eliglustat Tartrate – Powder for Oral Suspension1件: Eliglustat1件: Eliglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
202Elosulfase alfa1件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
203Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]1件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
204ELX-021件: ELX-02---3件: 19, 218, 299 💬
205Enalapril1件: Enalapril1件: Enalapril 💬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 💬
206Enalapril and other angiotensin converting enzyme inhibitors2件: Angiotensin II,
Enalapril		3件: Angiotensin II ,
Angiotensin II ,
Enalapril 💬		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 💬
207Enriched Hematopoetic Stem Cell Infusion----2件: 19, 284 💬
208Enzyme replacement----1件: 19 💬
209Enzyme replacement therapy----1件: 19 💬
210Enzyme replacement therapy (Agalsidase alpha (Replagal®))1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
211Enzyme replacement therapy (ERT)----1件: 19 💬
212ERT----1件: 19 💬
213EU-sourced Cerezyme1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
214FABRAZYME1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
215Fabrazyme (agalsidase beta)1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
216Fabrazyme (agalsidase beta) 1 mg/kg1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
217FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
218Fabrazyme 35 mg1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
219Fabrazyme 5 mg1件: Agalsidase beta1件: Agalsidase beta 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
220FBX-101----1件: 19 💬
221FLT190----1件: 19 💬
222FLT201----1件: 19 💬
223Fludarabina1件: Fludarabine1件: Fludarabine 💬1件: RRM1 💬6件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬2件: 19, 65 💬
224FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML1件: Fludarabine1件: Fludarabine 💬1件: RRM1 💬6件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬1件: 19 💬
225FLUDARABINA FOSFATO1件: Fludarabine1件: Fludarabine 💬1件: RRM1 💬6件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬1件: 19 💬
226Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the a-L-iduronidase cDNA, in their final formulation medium.----1件: 19 💬
227GA-GCB1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
228GA-GCB (velaglucerase alfa)1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
229GABAPENTIN1件: Gabapentin1件: Gabapentin 💬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 💬
230Galafold1件: Migalastat1件: Migalastat 💬--1件: 19 💬
231GALSULFASE1件: Galsulfase1件: Galsulfase 💬1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
232GC1111_0.5mg/kg----1件: 19 💬
233GC1111_1.0mg/kg----1件: 19 💬
234GC1119----1件: 19 💬
235GC1123----1件: 19 💬
236Gelatine1件: Gelatin2件: Gelatin ,
Donkey glue 💬		--1件: 19 💬
237Gene Activated a-Galactosidase A----1件: 19 💬
238Gene Activated Human Glucocerebrosidase----1件: 19 💬
239Gene activated human glucocerebrosidase, velaglucerase alfa1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
240Gene-Activated Human Glucocerebrosidase----1件: 19 💬
241Gene-Activated Human Glucocerebrosidase 200U/vial----1件: 19 💬
242Gene-Activated Human Glucocerebrosidase 400U/vial----1件: 19 💬
243Genetic analyses----1件: 19 💬
244Genetic HMI-203----1件: 19 💬
245GENISTEIN1件: Genistein1件: Genistein 💬--2件: 19, 299 💬
246Genistein aglycone1件: Genistein1件: Genistein 💬--1件: 19 💬
247Genz-112638----1件: 19 💬
248GENZ-6824521件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67 💬
249Genz-682452-AA1件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
250Genz-682452-AU1件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67 💬
251GLA----2件: 13, 19 💬
252GLA gene----1件: 19 💬
253GLA genetic sequencing.----1件: 19 💬
254GLUCOCEREBROSIDASE----1件: 19 💬
255GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO----1件: 19 💬
256GNR-055----1件: 19 💬
257GNR-055 1.0-2.0-3.0 mg/kg----1件: 19 💬
258GNR-055 2.0 mg/kg----1件: 19 💬
259GNR-055 3.0 mg/kg----1件: 19 💬
260GR181413----1件: 19 💬
261GR181413A----1件: 19 💬
262GR181413A/AT1001 capsule----1件: 19 💬
263GR181413A/AT1001 solution----1件: 19 💬
264Growth hormone1件: Somatotropin---14件: 2, 13, 19, 46, 78, 96, 107, 187, 191, 193, 195, 265, 276, 299 💬
265GSK2788723----1件: 19 💬
266GZ/SAR402671----2件: 19, 67 💬
267GZ385660----1件: 19 💬
268GZ402665----1件: 19 💬
269GZ402671----2件: 19, 67 💬
270GZ402671 / SAR402671----1件: 19 💬
271Hematopoetic Stem Cell Transplantation----5件: 19, 65, 120, 160, 326 💬
272Hematopoietic stem cell infusion----2件: 19, 20 💬
273Hematopoietic Stem Cell Transplantation----12件: 11, 13, 14, 19, 20, 35, 46, 49, 51, 96, 234, 285 💬
274Heparán N sulfatasa humana recombinante----1件: 19 💬
275Heparán N sulfatasa humana recombinante (rhHNS)----1件: 19 💬
276HGT-1110----1件: 19 💬
277HGT-1111----1件: 19 💬
278HGT-1410----1件: 19 💬
279HGT-2310----1件: 19 💬
280HIDS DONOR----1件: 19 💬
281HuCNS-SC----1件: 19 💬
282Human Glucocerebrosidase (prGCD)1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
283Human glucocerebrosidase cDNA----1件: 19 💬
284Human glucocerebrosidase gene into autologous peripheral blood stem cells----1件: 19 💬
285Human Placental Derived Stem Cell----4件: 19, 20, 60, 284 💬
286Hunterase1件: Idursulfase beta1件: Idursulfase beta 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
287Hydroxypropyl-beta-cyclodextrin1件: Betadex1件: Betadex 💬--1件: 19 💬
288Hydroxypropyl-ß-cyclodextrin----1件: 19 💬
289Hydroxyurea1件: Hydroxyurea1件: Hydroxyurea 💬1件: RRM2 💬7件: Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism, p53 signaling pathway 💬8件: 3, 13, 19, 20, 65, 85, 86, 284 💬
290I2S----1件: 19 💬
291I2S-IT----1件: 19 💬
292IB1001----2件: 19, 65 💬
293Iduronate 2-sulfatase1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
294Iduronate-2-sulfatase enzyme replacement therapy----1件: 19 💬
295IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR----1件: 19 💬
296IDURSULFASA1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
297IDURSULFASE1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
298Idursulfase (ELAPRASE)1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
299Idursulfase (I2S)1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
300Idursulfase (I2S)-IT1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
301Idursulfase beta2件: Idursulfase,
Idursulfase beta		2件: Idursulfase ,
Idursulfase beta 💬		1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
302Idursulfase IT (1 mg)1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
303Idursulfase IT (10 mg)1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
304Idursulfase IT (30 mg)1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
305Idursulfase(12S)-IT1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
306Idursulfase(I2S)-IT1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
307Idursulfase-IT1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
308IGF2----1件: 19 💬
309IMD----5件: 19, 20, 125, 234, 326 💬
310IMD Preparative Regimen----5件: 19, 20, 125, 234, 326 💬
311IMIGLUCERASA1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
312IMIGLUCERASE1件: Imiglucerase1件: Imiglucerase 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
313Imiglucerasi----1件: 19 💬
314Imiglucérase (drug) pharmacokinetics----1件: 19 💬
315Inactive Reminder Capsules----1件: 19 💬
316Inactive Reminder Product----1件: 19 💬
317INN Not available----2件: 19, 46 💬
318INNOZIDE----1件: 19 💬
319Intracerebral administration of AAVrh.10cuARSA----1件: 19 💬
320Intrathecal recombinant human alpha iduronidase----1件: 19 💬
321Iohexol1件: Iohexol1件: Iohexol 💬--2件: 19, 67 💬
322IRT Laronidase1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
323Isofagamine tartrate----1件: 19 💬
324ISU302----1件: 19 💬
325IV migalastat HCl1件: Migalastat1件: Migalastat 💬--1件: 19 💬
326IVA 336----1件: 19 💬
327JR-141----1件: 19 💬
328JR-141 or Idursulfase1件: Idursulfase1件: Idursulfase 💬1件: IDS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
329JR-171 (lepunafusp alfa)----1件: 19 💬
330KANUMA1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
331LAMAZYM1件: Velmanase alfa1件: Velmanase alfa 💬1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19 💬
332LARONIDASE1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬2件: 19, 256 💬
333Laronidase ERT1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
334Laronidase therapy and a stem cell transplant1件: Laronidase1件: Laronidase 💬1件: IDUA 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
335Ledipasvir1件: Ledipasvir1件: Ledipasvir 💬--1件: 19 💬
336Ledipasvir/Sofosbuvir2件: Ledipasvir,
Sofosbuvir		2件: Sofosbuvir ,
Ledipasvir 💬		--1件: 19 💬
337Lenograstim1件: Lenograstim1件: Lenograstim 💬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 💬
338Lentivirus Alpha-gal A transduced stem cells----1件: 19 💬
339Lentivirus-mediated delivery of ARSA to the CNS.----1件: 19 💬
340Leucine1件: Leucine1件: Leucine 💬--6件: 6, 19, 65, 283, 284, 299 💬
341Leucovorin1件: Leucovorin2件: Folinic acid ,
Folinic acid calcium salt pentahydrate 💬		--2件: 19, 46 💬
342Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA)----1件: 19 💬
343Lisinopril1件: Lisinopril2件: Lisinopril ,
Lisinopril 💬		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 💬
344Lopace----1件: 19 💬
345Losartan1件: Losartan1件: Losartan 💬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 💬17件: 17, 19, 36, 58, 66, 67, 85, 96, 97, 98, 113, 164, 167, 179, 215, 222, 299 💬
346Losartan and other angiotensin receptor blockers2件: Angiotensin II,
Losartan		3件: Angiotensin II ,
Angiotensin II ,
Losartan 💬		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 💬
347Lucerastat1件: Lucerastat1件: Lucerastat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
348LY38849611件: LY3884961---1件: 19 💬
349Lymphocyte gene therapy----1件: 19 💬
350LYS----1件: 19 💬
351LYS-GM101----1件: 19 💬
352LYS-SAF302----1件: 19 💬
353Lysodase----1件: 19 💬
354Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)1件: Cholesterol1件: Cholesterol 💬--1件: 19 💬
355Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)1件: Cholesterol1件: Cholesterol 💬--1件: 19 💬
356MabThera1件: Rituximab1件: Rituximab 💬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 💬
357MABTHERA - 2 FIALE 100 MG 10 ML1件: Rituximab1件: Rituximab 💬1件: MS4A1 💬1件: Hematopoietic cell lineage 💬2件: 19, 65 💬
358Medication to suppress the immune system----1件: 19 💬
359Melphalan1件: Melphalan1件: Melphalan 💬--15件: 11, 13, 14, 19, 20, 28, 60, 62, 65, 96, 164, 234, 284, 285, 299 💬
360Mepsevii1件: Vestronidase alfa1件: Vestronidase alfa 💬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 💬
361Mercaptamina1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
362Mercaptamine1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
363MERCAPTAMINE BITARTRATE1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
364MERCAPTAMINE HYDROCHLORIDE1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
365Mercaptamine, mercamine, 2-aminoethanethiol1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
366Metazym----1件: 19 💬
367Methotrexate1件: Methotrexate1件: Methotrexate 💬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 💬
368Methylprednisolone1件: Methylprednisolone1件: Methylprednisolone 💬1件: NR3C1 💬1件: Neuroactive ligand-receptor interaction 💬46件: 2, 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, 85, 86, 93, 95, 96, 97, 145, 162, 164, 211, 220, 222, 224, 271, 283, 284, 285, 296 💬
369MIGALASTAT1件: Migalastat1件: Migalastat 💬--1件: 19 💬
370Migalastat HCl1件: Migalastat1件: Migalastat 💬--1件: 19 💬
371Migalastat HCl 150 mg1件: Migalastat1件: Migalastat 💬--1件: 19 💬
372MIGALASTAT HYDROCHLORIDE1件: Migalastat1件: Migalastat 💬--1件: 19 💬
373Migalastat hydrocloride1件: Migalastat1件: Migalastat 💬--1件: 19 💬
374MIGLUSTAT1件: Miglustat1件: Miglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 19, 256, 299 💬
375MIGLUSTAT (ZAVESCA)1件: Miglustat1件: Miglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
376Moss-aGal (recombinant human alpha-galactosidase A produced in moss)1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
377Mozobil1件: Plerixafor1件: Plerixafor 💬1件: CXCR4 💬14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 life cycle - HIV-1, Viral protein interaction with cytokine and cytokine receptor, Virion - Human immunodeficiency virus 💬3件: 19, 65, 285 💬
378MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F1件: Plerixafor1件: Plerixafor 💬1件: CXCR4 💬14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 life cycle - HIV-1, Viral protein interaction with cytokine and cytokine receptor, Virion - Human immunodeficiency virus 💬1件: 19 💬
379Mucoangin----1件: 19 💬
380Mycophenolate1件: Mycophenolic acid1件: Mycophenolic acid 💬2件: IMPDH1,
IMPDH2 💬		4件: Drug metabolism - other enzymes, Metabolic pathways, Nucleotide metabolism, 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 💬
381Mycophenolate mofetil2件: Mycophenolate mofetil,
Mycophenolic acid		2件: Mycophenolate mofetil ,
Mycophenolic acid 💬		2件: IMPDH1,
IMPDH2 💬		4件: Drug metabolism - other enzymes, Metabolic pathways, Nucleotide metabolism, 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 💬
382Mycophenylate mofetil----2件: 19, 20 💬
383MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML----2件: 19, 65 💬
384N acetyl cysteine1件: Cysteine---1件: 19 💬
385N Acetyl L Leucine1件: Leucine1件: Leucine 💬--1件: 19 💬
386N-Acetyl-L Leucine1件: Leucine1件: Leucine 💬--1件: 19 💬
387N-Acetyl-L-Leucine2件: L-Acetylleucine,
Leucine		1件: Leucine 💬--2件: 19, 65 💬
388N-Acetylcysteine1件: Acetylcysteine1件: Acetylcysteine 💬--13件: 6, 13, 19, 20, 21, 49, 58, 85, 90, 111, 164, 299, 337 💬
389N-acetylgalactosamine 4-sulfatase----1件: 19 💬
390Naglazyme1件: Galsulfase1件: Galsulfase 💬1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
391Naglazyme (galsulfase)1件: Galsulfase1件: Galsulfase 💬1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
392Naglazyme®1件: Galsulfase1件: Galsulfase 💬1件: ARSB 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
393NGN-101----1件: 19 💬
394Non applicable----2件: 10, 19 💬
395Odiparcil1件: Odiparcil---1件: 19 💬
396OGT 918----2件: 19, 299 💬
397OGT918----1件: 19 💬
398OGT923----1件: 19 💬
399OLENASUFLIGENE RELDUPARVOVEC1件: Olenasufligene relduparvovec---1件: 19 💬
400OLIPUDASE ALFA1件: Olipudase alfa1件: Olipudase alfa 💬1件: SMPD1 💬5件: Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬1件: 19 💬
401Olipudase alfa (GZ402665)1件: Olipudase alfa1件: Olipudase alfa 💬1件: SMPD1 💬5件: Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬1件: 19 💬
402Olipudase alfa (rhASM)1件: Olipudase alfa1件: Olipudase alfa 💬1件: SMPD1 💬5件: Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬1件: 19 💬
403OMNIPAQUE1件: Iohexol1件: Iohexol 💬--2件: 19, 222 💬
404Oral migalastat HCl1件: Migalastat1件: Migalastat 💬--1件: 19 💬
405Osteopetrosis Haploidentical Only Preparative Regimen----5件: 19, 20, 125, 234, 326 💬
406Osteopetrosis Only Preparative Regimen----5件: 19, 20, 125, 234, 326 💬
407OTL-200----1件: 19 💬
408OTL-200 Dispersion for Infusion----1件: 19 💬
409OTL-200 Gene Therapy----1件: 19 💬
410Pabinafusp alfa1件: Pabinafusp alfa1件: Pabinafusp alfa 💬2件: IDS,
TFRC 💬		8件: Endocytosis, Ferroptosis, Glycosaminoglycan degradation, HIF-1 signaling pathway, Hematopoietic cell lineage, Lysosome, Metabolic pathways, Phagosome 💬1件: 19 💬
411PARACETAMOLO----9件: 2, 11, 14, 19, 28, 53, 61, 63, 300 💬
412PARACETAMOLO DOC GENERICI - 1000 MG COMPRESSA 16 COMPRESSE----1件: 19 💬
413Paricalcitol1件: Paricalcitol1件: Paricalcitol 💬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 💬5件: 19, 66, 218, 238, 298 💬
414PBGM01----1件: 19 💬
415PBKR03----1件: 19 💬
416PEGUNIGALSIDASE ALFA1件: Pegunigalsidase alfa1件: Pegunigalsidase alfa 💬--1件: 19 💬
417Pegunigalsidase alpha----1件: 19 💬
418Pending----6件: 13, 19, 96, 97, 113, 302 💬
419Pentosan Polysulfat SP 54® injection solution----1件: 19 💬
420Pentosan polysulfate1件: Pentosan polysulfate---3件: 19, 26, 226 💬
421PENTOSAN POLYSULFATE SODIUM1件: Pentosan polysulfate---1件: 19 💬
422Pentosan Polysulphate SP54----1件: 19 💬
423Pentosan Polysulphate SP54®----1件: 19 💬
424Perdix----1件: 19 💬
425Plant cell expressed recombinant glucocerebrosidase (prGCD)1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
426Plant Cell Recombinant Glucocerebrosidase----1件: 19 💬
427Plasma Lyso-GB3----1件: 19 💬
428PLX-200----1件: 19 💬
429Prednisone1件: Prednisone1件: Prednisone 💬1件: NR3C1 💬1件: Neuroactive ligand-receptor interaction 💬45件: 2, 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, 162, 164, 205, 222, 224, 228, 283, 284, 288, 299, 300, 307, 331 💬
430PREDNISONE DOC GENERICI - 5 MG COMPRESSE 10 COMPRESSE IN BLISTER PVC-PVDC/ALU1件: Prednisone1件: Prednisone 💬1件: NR3C1 💬1件: Neuroactive ligand-receptor interaction 💬1件: 19 💬
431Prevenar13----2件: 19, 265 💬
432PrGCD1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
433PROCYSBI1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
434PRX-102----1件: 19 💬
435PRX-102 (pegunigalsidase alfa)1件: Pegunigalsidase alfa1件: Pegunigalsidase alfa 💬--1件: 19 💬
436PRX-102 1 mg/kg every 2 weeks----1件: 19 💬
437PRX-102 2 mg/kg every 4 weeks----1件: 19 💬
438PRX-112----1件: 19 💬
439PRX102----1件: 19 💬
440PTC124----4件: 19, 113, 246, 299 💬
441Purified form of the lysosomal enzyme iduronate-2-sulfatase----1件: 19 💬
442PYRIMETHAMINE1件: Pyrimethamine1件: Pyrimethamine 💬--4件: 2, 4, 19, 65 💬
443RAAV 2/6 hIDS DONOR----1件: 19 💬
444RAAV2/5-hNAGLU----1件: 19 💬
445RAAV2/6 Left ZFN Vector----1件: 19 💬
446RAAV2/6 Right ZFN Vector----1件: 19 💬
447RAAV9.CMV.hNAGLU----1件: 19 💬
448Rebisufligene etisparvovec1件: Rebisufligene etisparvovec---1件: 19 💬
449Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A----1件: 19 💬
450Recombinant alpha-galactosidase A1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
451Recombinant human acid sphingomyelinase----1件: 19 💬
452RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)----1件: 19 💬
453Recombinant human alpha-galactosidase----1件: 19 💬
454Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL----1件: 19 💬
455Recombinant human alpha-mannosidase----1件: 19 💬
456Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)----1件: 19 💬
457RECOMBINANT HUMAN ARYLSULFATASE A----1件: 19 💬
458Recombinant Human Arylsulfatase A (rhASA)----1件: 19 💬
459Recombinant Human Arylsulfatase A (rhASA,----1件: 19 💬
460Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase----1件: 19 💬
461RECOMBINANT HUMAN BETA GLUCURONIDASE----1件: 19 💬
462Recombinant human beta-glucuronidase1件: Vestronidase alfa1件: Vestronidase alfa 💬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 💬
463Recombinant human beta-glucuronidase (rhGUS)1件: Vestronidase alfa1件: Vestronidase alfa 💬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 💬
464Recombinant human betaglucuronidase----1件: 19 💬
465Recombinant human derived macrophage-targeted beta-Glucocerebrosidase----1件: 19 💬
466Recombinant human derived macrophage-targeted ß-Glucocerebrosidase----1件: 19 💬
467Recombinant human glucerebrosidase----1件: 19 💬
468Recombinant human glucocerebrosidase----1件: 19 💬
469Recombinant Human Glucocerebrosidase (prGCD)1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
470Recombinant human heparan N-sulfatase----1件: 19 💬
471Recombinant human heparan N-sulfatase (rhHNS)----1件: 19 💬
472Recombinant human heparan N-sulfatase [rhHNS]----1件: 19 💬
473Recombinant human heparan Nsulfatase (rhHNS)----1件: 19 💬
474Recombinant human lysosomal acid lipase----1件: 19 💬
475Recombinant human lysosomal acid lipase (rhLAL)----1件: 19 💬
476Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)1件: Cholesterol1件: Cholesterol 💬--1件: 19 💬
477Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA)1件: Cholesterol1件: Cholesterol 💬--1件: 19 💬
478Recombinant human lysosomal alpha-mannosidase----1件: 19 💬
479Recombinant human N-acetylgalactosamine 4-sulfatase----1件: 19 💬
480Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110----1件: 19 💬
481Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance----1件: 19 💬
482Recombinant human N-acetylgalactosamine-6-sulfatase1件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
483Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)1件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
484Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 1101件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
485Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance1件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
486Recombinant human Nacetylgalactosamine-----1件: 19 💬
487Recombinant human tripeptidyl peptidase-1----1件: 19 💬
488RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)----1件: 19 💬
489Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene----1件: 19 💬
490Reduced Intensity Conditioning----2件: 19, 65 💬
491Reminder capsules----1件: 19 💬
492Reminder Product----1件: 19 💬
493REPLAGAL1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
494Replagal (Agalsidase Alfa)1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
495Replagal agalsidase alfa1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
496REPLAGAL ®1件: Agalsidase alfa1件: Agalsidase alfa 💬1件: GLA 💬6件: Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
497RGX-111----1件: 19 💬
498RGX-121----1件: 19 💬
499Rh Growth Hormone1件: Somatotropin---1件: 19 💬
500RhARSA----1件: 19 💬
501RhASA----1件: 19 💬
502RhASA - Dose Level 1----1件: 19 💬
503RhASA - Dose Level 2----1件: 19 💬
504RhASA - Dose Level 3----1件: 19 💬
505RhASM----1件: 19 💬
506RhGALNS1件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
507RHGUS----1件: 19 💬
508RhHNS-10 mg----1件: 19 💬
509RhHNS-45 mg----1件: 19 💬
510RhHNS-90 mg----1件: 19 💬
511RhIDU (recombinant human-Alpha-L-Iduronidase)----1件: 19 💬
512RhLAMAN1件: Velmanase alfa1件: Velmanase alfa 💬1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19 💬
513RhNAGLU----1件: 19 💬
514RhNAGLU radiolabelled----1件: 19 💬
515RHNAGLU-IGF2----1件: 19 💬
516Rimiducid1件: Rimiducid1件: Rimiducid 💬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 💬
517RITUXIMAB1件: Rituximab1件: Rituximab 💬1件: MS4A1 💬1件: Hematopoietic cell lineage 💬48件: 11, 13, 14, 19, 26, 35, 36, 40, 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, 162, 222, 229, 256, 271, 274, 283, 284, 285, 288, 296, 300, 331 💬
518Rivogenlecleucel----4件: 19, 60, 285, 326 💬
519RP 103----2件: 8, 19 💬
520RP103----2件: 8, 19 💬
521RVX000222----1件: 19 💬
522SAF-301----1件: 19 💬
523SAR402671, GZ402671 o GZ/SAR402671----2件: 19, 67 💬
524SAR402671, GZ402671 or GZ / SAR402671----1件: 19 💬
525SAR402671, GZ402671 or GZ/SAR402671----3件: 6, 19, 67 💬
526SB-318----1件: 19 💬
527SB-47171----1件: 19 💬
528SB-47898----1件: 19 💬
529SB-913----1件: 19 💬
530SB-A6P-HRL Donor Vector----1件: 19 💬
531SB-FIX----1件: 19 💬
532SB-IDUA----1件: 19 💬
533SBC-1021件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
534SBC-102, recombinant human lysosomal acid lipase1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
535SBC-103----1件: 19 💬
536ScAAV9.CB.CLN6----1件: 19 💬
537ScAAV9.U1A.hSGSH----1件: 19 💬
538ScAAV9.U1A.SGSH----1件: 19 💬
539Sebelipasa Alfa1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
540Sebelipase Alfa1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
541Sebelipase alfa (SBC-102)1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
542Sebelipase alfa 0.35 mg/kg1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
543Sebelipase alfa 1 mg/kg1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
544Sebelipase alfa 3 mg/kg1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
545SHP611----1件: 19 💬
546SHP675----1件: 19 💬
547Sirolimus1件: Sirolimus1件: Sirolimus 💬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 💬35件: 13, 15, 17, 19, 34, 35, 36, 46, 49, 51, 60, 61, 62, 63, 65, 66, 67, 84, 85, 86, 89, 98, 137, 157, 158, 192, 222, 277, 278, 279, 280, 281, 283, 300, 331 💬
548SOBI003----1件: 19 💬
549Sofosbuvir1件: Sofosbuvir1件: Sofosbuvir 💬--1件: 19 💬
550Somatropin1件: Somatotropin---19件: 2, 3, 19, 46, 65, 74, 75, 78, 96, 107, 113, 187, 193, 195, 238, 265, 274, 276, 299 💬
551Somatropin (DNA origin)1件: Somatotropin---1件: 19 💬
552Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride1件: Cysteamine1件: Cysteamine 💬--1件: 19 💬
553ST-920----1件: 19 💬
554Stem Cell Transplantation----10件: 13, 19, 20, 46, 65, 90, 125, 164, 234, 326 💬
555SUB195712----1件: 19 💬
556TALIGLUCERASE ALFA1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
557Taliglucerase alfa - Recombinant human glucocerebrosidase1件: Taliglucerase alfa1件: Taliglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
558TBD----5件: 19, 86, 90, 276, 299 💬
559Thiotepa1件: Thiotepa1件: Thiotepa 💬--12件: 13, 19, 20, 28, 51, 60, 65, 85, 96, 164, 284, 285 💬
560Thiotepa--escalated dose1件: Thiotepa1件: Thiotepa 💬--4件: 19, 20, 65, 284 💬
561Thiotepa--single daily dose1件: Thiotepa1件: Thiotepa 💬--4件: 19, 20, 65, 284 💬
562Thyroxine1件: Levothyroxine1件: Levothyroxine 💬2件: THRA,
THRB 💬		2件: Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬1件: 19 💬
563Tralesinidase alfa----1件: 19 💬
564Transduced CD34+ hematopoietic stem cell----2件: 19, 20 💬
565Trappsol Cyclo----1件: 19 💬
566TREOSULFAN1件: Treosulfan1件: Treosulfan 💬--6件: 13, 19, 62, 65, 283, 286 💬
567Triapin----1件: 19 💬
568Triapin& triapin Mite----1件: 19 💬
569TSHA-101----1件: 19 💬
570Unrelated Umbilical Cord Blood Transplant----1件: 19 💬
571UPLYSO----1件: 19 💬
572USAN: sebelipasa alfa1件: Sebelipase alfa1件: Sebelipase alfa 💬1件: LIPA 💬3件: Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬1件: 19 💬
573USAN: sebelipase alpha----1件: 19 💬
574UX003----1件: 19 💬
575Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana----1件: 19 💬
576VELAGLUCERASE ALFA1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
577Velaglucerasi alfa----1件: 19 💬
578VELMANASE ALFA1件: Velmanase alfa1件: Velmanase alfa 💬1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19 💬
579Velmanase Alfa (e.g. Lamazym)1件: Velmanase alfa1件: Velmanase alfa 💬1件: MAN2B1 💬2件: Lysosome, Other glycan degradation 💬1件: 19 💬
580Venglustat1件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67 💬
581Venglustat (GZ402671)1件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
582Venglustat GZ4026711件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬2件: 19, 67 💬
583Venglustat malate1件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 6, 19, 67 💬
584Venglustat malato1件: Venglustat1件: Venglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬2件: 19, 67 💬
585Vestronidase alfa1件: Vestronidase alfa1件: Vestronidase alfa 💬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 💬
586Vimizim® (elosulfase alfa)1件: Elosulfase alfa1件: Elosulfase alfa 💬1件: GALNS 💬3件: Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬1件: 19 💬
587Vitamin D2件: Cholecalciferol,
Vitamin D		1件: Cholecalciferol 💬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 💬20件: 13, 19, 41, 43, 44, 46, 49, 50, 68, 93, 95, 96, 97, 179, 222, 228, 235, 274, 298, 299 💬
588VPRIV1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
589VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
590VPRIV 400 Units powder for solution of infusion1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
591VPRIV ®,1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
592VPRIV®1件: Velaglucerase alfa1件: Velaglucerase alfa 💬1件: GBA1 💬4件: Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬1件: 19 💬
593VTS-270----1件: 19 💬
594Warfarin1件: Warfarin1件: Warfarin 💬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 💬11件: 6, 8, 13, 19, 46, 49, 66, 85, 86, 88, 91 💬
595ZAVESCA1件: Miglustat1件: Miglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬3件: 19, 256, 299 💬
596Zavesca (Miglustat)1件: Miglustat1件: Miglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
597ZAVESCA 84CPS 100MG1件: Miglustat1件: Miglustat 💬1件: UGCG 💬2件: Metabolic pathways, Sphingolipid metabolism 💬1件: 19 💬
598Zestoretic 10,201件: Lisinopril2件: Lisinopril ,
Lisinopril 💬		1件: ACE 💬6件: Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬1件: 19 💬
599ZESTORIC----1件: 19 💬
600Zestril1件: Lisinopril2件: Lisinopril ,
Lisinopril 💬		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 💬