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No. Drug (Description in trials) DrugBank KEGG DRUG KEGG GENES KEGG PATHWAY Disease ID
1045251016----[1] 19 💬
21- Deoxygalactonojirimycin----[1] 19 💬
31-Deoxygalactonojirimycin[1] Migalastat[1] Migalastat 💬--[1] 19 💬
41-deoxygalactonojirimycin hydrochloride[1] Migalastat[1] Migalastat 💬--[1] 19 💬
512S-IT----[1] 19 💬
615-0 H20----[2] 6, 19 💬
72(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID----[2] 19, 65 💬
82-aminoethanethiol[1] Cysteamine[1] Cysteamine 💬--[2] 19, 299 💬
92-HYDROXYPROPYL-BETA-CYCLODEXTRIN[2] Betadex,
Hydroxypropyl betadex		[1] Betadex 💬--[1] 19 💬
1026 common Fabry mutation types in Taiwan----[1] 19 💬
114D-310----[1] 19 💬
12500 0500 0----[1] 19 💬
1389 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)----[1] 19 💬
1489Zr-SBC-103----[1] 19 💬
15[14C] AT1001----[1] 19 💬
16A (migalastat)[1] Migalastat[1] Migalastat 💬--[1] 19 💬
17A16AB04----[1] 19 💬
18A16AX06----[1] 19 💬
19AAV2/8.TBG.hARSB----[1] 19 💬
20AAV2CUhCLN2 (3x10^12 particle units)----[1] 19 💬
21AAV9-CAG-coh-SGSH----[1] 19 💬
22AAV9-GLB1----[1] 19 💬
23AAVrh.10-hMPSIIIA----[1] 19 💬
24AAVrh.10cuARSA----[1] 19 💬
25AAVrh.10CUCLN2----[1] 19 💬
26AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies----[1] 19 💬
27AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies----[1] 19 💬
28Abatacept[1] Abatacept[1] Abatacept 💬[2] CD80,
CD86 💬		[13] 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] 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] 19 💬
30ABO-101----[1] 19 💬
31ABO-102----[1] 19 💬
32Accupro----[1] 19 💬
33Accuretic[1] Quinapril[1] Quinapril 💬[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[1] 19 💬
34Acetyl-l-leucine[2] L-Acetylleucine,
Leucine		[1] Leucine 💬--[2] 19, 65 💬
35Acetylcysteine[1] Acetylcysteine[1] Acetylcysteine 💬--[21] 6, 13, 19, 20, 21, 49, 51, 53, 58, 63, 85, 90, 94, 111, 164, 193, 233, 296, 298, 299, 337 💬
36ACT-434964----[1] 19 💬
37Adalimumab[1] Adalimumab[1] Adalimumab 💬[1] TNF 💬[67] 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] 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 GENE[1] Levothyroxine[1] Levothyroxine 💬[2] THRA,
THRB 💬		[2] Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬[1] 19 💬
39Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene----[1] 19 💬
40Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-betagal)----[1] 19 💬
41Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal)----[1] 19 💬
42Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
43Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
44Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector----[1] 19 💬
45Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector----[1] 19 💬
46Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector----[1] 19 💬
47Adeno-associated virus serotype hu68 containing the human GALC gene----[1] 19 💬
48Adeno-associated virus serotype rh.10 expressing beta-galactosidase[1] Tilactase[1] Lactase 💬--[1] 19 💬
49ADMINISTRATION----[1] 19 💬
50Adrabetadex[1] Adrabetadex---[1] 19 💬
51Afegostat[1] Afegostat[1] Afegostat 💬--[1] 19 💬
52Afegostat tartrate[1] Afegostat[1] Afegostat 💬--[1] 19 💬
53Agalsidase----[1] 19 💬
54AGALSIDASE ALFA[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
55Agalsidase Alpha[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
56AGALSIDASE BETA[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
57Agalsidase beta (GZ419828)[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
58Agalsidase beta (recombinant form)[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
59Agalsidase beta from Biosidus 1 mg/kg[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
60AGALSIDASI ALFA----[1] 19 💬
61AGALSIDASI BETA----[1] 19 💬
62AGT-181----[1] 19 💬
63AGT-181 (HIRMAb-IDUA)----[1] 19 💬
64AGT-182----[1] 19 💬
65Aldurazyme[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
66Aldurazyme (laronidase)[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[2] 19, 256 💬
67Aldurazyme (Recombinant Human Alpha-L-Iduronidase)[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
68ALDURAZYME*INFUS 1FL 5ML 500U[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
69ALEMTUZUMAB[1] Alemtuzumab[1] Alemtuzumab 💬[1] CD52 💬-[17] 13, 15, 19, 20, 36, 43, 44, 46, 51, 60, 63, 65, 85, 96, 164, 283, 284 💬
70Alendronate[1] Alendronic acid[1] Alendronic acid 💬[1] FDPS 💬[4] Human T-cell leukemia virus 1 infection, Influenza A, Metabolic pathways, Terpenoid backbone biosynthesis 💬[7] 19, 41, 46, 50, 271, 274, 299 💬
71Alendronate sodium[1] Alendronic acid[1] Alendronic acid 💬[1] FDPS 💬[4] Human T-cell leukemia virus 1 infection, Influenza A, Metabolic pathways, Terpenoid backbone biosynthesis 💬[2] 19, 299 💬
72Alfa-manosidadas humana recombinante----[1] 19 💬
73ALISERIN - 25 MG GRANULATO EFFERVESCENTE 20 BUSTINE----[1] 19 💬
74Alpha-galactosidase A[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
75Ambroxol[1] Ambroxol[1] Ambroxol 💬--[2] 6, 19 💬
76Ambroxol Hydrochloride[1] Ambroxol[1] Ambroxol 💬--[2] 6, 19 💬
77AMBROXOL HYDROCHLORIDE PH. EUR.[1] Ambroxol[1] Ambroxol 💬--[1] 19 💬
78Angiotensin[1] Angiotensin II[2] Angiotensin II ,
Angiotensin II 💬		[2] AGTR1,
AGTR2 💬		[16] 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] 17, 19, 46, 66, 86, 222, 224 💬
79ARIMOCLOMOL[1] Arimoclomol[1] Arimoclomol 💬--[3] 2, 15, 19 💬
80Arimoclomol (hard gelatine capsule)[2] Arimoclomol,
Gelatin		[3] Gelatin ,
Donkey glue ,
Arimoclomol 💬		--[1] 19 💬
81Arimoclomol (HPMC capsule)[1] Arimoclomol[1] Arimoclomol 💬--[1] 19 💬
82Arimoclomol citrate[1] Arimoclomol[1] Arimoclomol 💬--[2] 15, 19 💬
83Arylsulfatase A----[1] 19 💬
84AT-GTX-501----[1] 19 💬
85AT1001----[1] 19 💬
86AT1001 150 mg----[1] 19 💬
87AT2101----[1] 19 💬
88ATALUREN[1] Ataluren[1] Ataluren 💬--[6] 19, 113, 140, 246, 299, 329 💬
89AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE----[1] 19 💬
90Autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA----[1] 19 💬
91Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII----[1] 19 💬
92Autologous Plasmablasts----[1] 19 💬
93Autologous Plasmablasts (B cells)----[1] 19 💬
94AVR-RD-01----[1] 19 💬
95AVR-RD-02----[1] 19 💬
96AX----[1] 19 💬
97AX 250----[1] 19 💬
98AXO-AAV-GM2 High Dose----[1] 19 💬
99AXO-AAV-GM2 Low Dose----[1] 19 💬
100AXO-AAV-GM2 Middle Dose----[1] 19 💬
101AXO-AAV-GM2 Starting Dose----[1] 19 💬
102Azathioprine[1] Azathioprine[1] Azathioprine 💬--[21] 6, 11, 13, 19, 35, 42, 43, 44, 45, 46, 49, 63, 65, 66, 85, 93, 94, 95, 96, 97, 162 💬
103Azathioprine (AZA)[1] Azathioprine[1] Azathioprine 💬--[3] 13, 19, 97 💬
104B (migalastat)[1] Migalastat[1] Migalastat 💬--[1] 19 💬
105Beta-cyclodextrin[1] Betadex[1] Betadex 💬--[1] 19 💬
106Beta-Galactosidase[1] Tilactase[1] Lactase 💬--[1] 19 💬
107Beta-Mercaptoethylamine[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
108Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
109BHP001----[1] 19 💬
110Blood sampling for biological and genetic analysis----[1] 19 💬
111BM110----[1] 19 💬
112BMN 110----[1] 19 💬
113BMN 110 - Every Other Week----[1] 19 💬
114BMN 110 - Weekly----[1] 19 💬
115BMN 110 Every Other Week----[1] 19 💬
116BMN 110 Weekly----[1] 19 💬
117BMN 190----[1] 19 💬
118BMN 250----[1] 19 💬
119BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)----[1] 19 💬
120Brineura[1] Cerliponase alfa[1] Cerliponase alfa 💬[1] TPP1 💬[1] Lysosome 💬[1] 19 💬
121BUSILVEX[1] Busulfan[1] Busulfan 💬--[4] 19, 36, 60, 65 💬
122BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI[1] Busulfan[1] Busulfan 💬--[2] 19, 65 💬
123Busulfan[1] Busulfan[1] Busulfan 💬--[13] 19, 20, 28, 36, 49, 51, 60, 65, 164, 274, 284, 285, 326 💬
124Busulfan, Cyclophosphamide, Antithymocyte Globulin[2] Busulfan,
Cyclophosphamide		[3] Busulfan ,
Cyclophosphamide ,
Cyclophosphamide 💬		--[2] 19, 20 💬
125Busulfan, Cyclophosphamide, ATG[2] Busulfan,
Cyclophosphamide		[3] Busulfan ,
Cyclophosphamide ,
Cyclophosphamide 💬		--[1] 19 💬
126BUSULFANO[1] Busulfan[1] Busulfan 💬--[1] 19 💬
127C (migalastat)[1] Migalastat[1] Migalastat 💬--[1] 19 💬
128Calcium[2] Calcium,
Calcium carbonate		[2] Calcium carbonate ,
Calcium carbonate 💬		--[27] 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 carbonate[3] Calcium,
Calcium carbonate,
Carbonate ion		[2] Calcium carbonate ,
Calcium carbonate 💬		--[8] 13, 19, 46, 53, 75, 96, 235, 299 💬
130CALD HR-D (High-Risk, Regimen C)----[5] 19, 20, 125, 234, 326 💬
131CALD HR-D (High-Risk, Regimen D)----[5] 19, 20, 125, 234, 326 💬
132CALD SR-A (Standard-Risk, Regimen A)----[5] 19, 20, 125, 234, 326 💬
133CALD SR-B (Standard-Risk, Regimen B)----[5] 19, 20, 125, 234, 326 💬
134Campath[1] Alemtuzumab[1] Alemtuzumab 💬[1] CD52 💬-[12] 19, 20, 49, 51, 60, 62, 65, 164, 234, 283, 285, 326 💬
135Campath-1H[1] Alemtuzumab[1] Alemtuzumab 💬[1] CD52 💬-[6] 19, 20, 60, 234, 285, 326 💬
136Cannabis[1] Medical Cannabis---[7] 2, 6, 13, 19, 90, 96, 97 💬
137Cannabis sativa L., folium cum flore[1] Medical Cannabis---[1] 19 💬
138Carbonate[1] Carbonate ion---[12] 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] 19 💬
140Cerdelga[1] Eliglustat[1] Eliglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
141Ceredase[1] Alglucerase[1] Alglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
142CEREDASE™[1] Alglucerase[1] Alglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
143CEREZYME[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
144Cerezyme (imiglucerase for injection)[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
145Cerezyme 400 Units Powder for concentrate for solution for infusion[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
146Cerezyme®[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
147Cerezyme® / Imiglucerase[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
148Cerliponase Alfa[1] Cerliponase alfa[1] Cerliponase alfa 💬[1] TPP1 💬[1] Lysosome 💬[1] 19 💬
149CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE----[1] 19 💬
150CHF6657----[1] 19 💬
151CHOLECALCIFEROL[1] Cholecalciferol[1] Cholecalciferol 💬[1] VDR 💬[5] Chemical carcinogenesis - receptor activation, Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis 💬[9] 13, 19, 34, 46, 49, 96, 97, 298, 299 💬
152Cholesterol[1] Cholesterol[1] Cholesterol 💬--[6] 8, 19, 90, 160, 222, 310 💬
153Clofarabine[1] Clofarabine[1] Clofarabine 💬[3] POLA1,
POLA2,
RRM1 💬		[7] DNA replication, Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬[5] 19, 20, 60, 234, 326 💬
154Coversyl[1] Perindopril[1] Perindopril 💬[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[2] 19, 167 💬
155Coversyl PLUS----[1] 19 💬
156CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE----[1] 19 💬
157CsA----[8] 19, 49, 50, 53, 60, 63, 222, 228 💬
158CTNS-RD-04 or CTNS-RD-04-LB (where the suffix -LB stands for LentiBOOST)----[1] 19 💬
159Cyclophosphamide[1] Cyclophosphamide[2] Cyclophosphamide ,
Cyclophosphamide 💬		--[44] 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 💬
160Cyclosporine[1] Cyclosporine[1] Cyclosporine 💬[5] PPP3CA,
PPP3CB,
PPP3CC,
PPP3R1,
PPP3R2 💬		[33] 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] 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 A[1] Cyclosporine[1] Cyclosporine 💬[5] PPP3CA,
PPP3CB,
PPP3CC,
PPP3R1,
PPP3R2 💬		[33] 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] 19, 20, 36, 49, 50, 53, 58, 60, 107, 234, 283 💬
162Cyclosporine A (CsA)[1] Cyclosporine[1] Cyclosporine 💬[5] PPP3CA,
PPP3CB,
PPP3CC,
PPP3R1,
PPP3R2 💬		[33] 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] 19, 60 💬
163Cystadane[1] Betaine[1] Betaine 💬--[1] 19 💬
164Cystadane anhydrous[1] Betaine[1] Betaine 💬--[1] 19 💬
165CYSTADROPS[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
166CYSTADROPS 0.55% eye drops, solution[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
167CYSTAGON[1] Cysteamine[1] Cysteamine 💬--[3] 8, 19, 299 💬
168Cystagon 150mg[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
169Cystagon 50mg[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
170Cystagon®[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
171Cystagon® (Cysteamine Bitartrate)[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
172Cysteamine[1] Cysteamine[1] Cysteamine 💬--[4] 8, 19, 21, 299 💬
173Cysteamine (mercaptamine) viscous solution[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
174Cysteamine Bitartrate[1] Cysteamine[1] Cysteamine 💬--[4] 8, 19, 21, 299 💬
175Cysteamine bitartrate (INN: mercaptamine bitartrate[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
176Cysteamine bitartrate (INN: mercaptamine bitartrate )[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
177Cysteamine bitartrate (INN: mercaptamine bitartrate)[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
178Cysteamine Bitartrate Delayed-release Capsules[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
179Cysteamine Bitartrate Delayed-release Capsules (RP103)[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
180CYSTEAMINE HYDROCHLORIDE[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
181Cysteine[1] Cysteine---[10] 13, 19, 34, 84, 85, 88, 90, 254, 296, 310 💬
182D (migalastat)[1] Migalastat[1] Migalastat 💬--[1] 19 💬
183Dapagliflozin[1] Dapagliflozin[1] Dapagliflozin 💬[1] SLC5A2 💬-[7] 19, 49, 66, 67, 86, 88, 222 💬
184Dapagliflozin 10mg Tab[1] Dapagliflozin[1] Dapagliflozin 💬[1] SLC5A2 💬-[1] 19 💬
185Diagnosis of Fabry disease----[1] 19 💬
186DIFENIDRAMINA CLORIDRATO----[3] 11, 19, 63 💬
187DNL310----[1] 19 💬
188DNL310 Drug Substance----[1] 19 💬
189Dried blood spot (DBS) sampling----[1] 19 💬
190DRX005B----[1] 19 💬
191DUOC-01----[3] 13, 19, 20 💬
192E (migalastat)[1] Migalastat[1] Migalastat 💬--[1] 19 💬
193Elaprase[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
194Elaprase 2mg/ml[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
195Elaprase for intravenous (IV) infusion[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
196Elaprase_0.5mg/kg----[1] 19 💬
197Elelyso[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
198Elelyso 60 units/kg[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
199Eliglustat[1] Eliglustat[1] Eliglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
200ELIGLUSTAT TARTRATE[1] Eliglustat[1] Eliglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
201Eliglustat Tartrate – Powder for Oral Suspension[1] Eliglustat[1] Eliglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
202Elosulfase alfa[1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
203Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM][1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
204ELX-02[1] ELX-02---[3] 19, 218, 299 💬
205Enalapril[1] Enalapril[1] Enalapril 💬[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[6] 19, 46, 66, 113, 210, 222 💬
206Enalapril and other angiotensin converting enzyme inhibitors[2] Angiotensin II,
Enalapril		[3] Angiotensin II ,
Angiotensin II ,
Enalapril 💬		[3] ACE,
AGTR1,
AGTR2 💬		[18] 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] 19 💬
207Enriched Hematopoetic Stem Cell Infusion----[2] 19, 284 💬
208Enzyme replacement----[1] 19 💬
209Enzyme replacement therapy----[1] 19 💬
210Enzyme replacement therapy (Agalsidase alpha (Replagal®))[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
211Enzyme replacement therapy (ERT)----[1] 19 💬
212ERT----[1] 19 💬
213EU-sourced Cerezyme[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
214FABRAZYME[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
215Fabrazyme (agalsidase beta)[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
216Fabrazyme (agalsidase beta) 1 mg/kg[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
217FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
218Fabrazyme 35 mg[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
219Fabrazyme 5 mg[1] Agalsidase beta[1] Agalsidase beta 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
220FBX-101----[1] 19 💬
221FLT190----[1] 19 💬
222FLT201----[1] 19 💬
223Fludarabina[1] Fludarabine[1] Fludarabine 💬[1] RRM1 💬[6] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬[2] 19, 65 💬
224FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML[1] Fludarabine[1] Fludarabine 💬[1] RRM1 💬[6] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬[1] 19 💬
225FLUDARABINA FOSFATO[1] Fludarabine[1] Fludarabine 💬[1] RRM1 💬[6] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism 💬[1] 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] 19 💬
227GA-GCB[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
228GA-GCB (velaglucerase alfa)[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
229GABAPENTIN[1] Gabapentin[1] Gabapentin 💬[5] CACNA2D1,
CACNA2D2,
CACNA2D3,
CACNA2D4,
SLC6A1 💬		[9] 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] 6, 13, 19, 70, 298 💬
230Galafold[1] Migalastat[1] Migalastat 💬--[1] 19 💬
231GALSULFASE[1] Galsulfase[1] Galsulfase 💬[1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
232GC1111_0.5mg/kg----[1] 19 💬
233GC1111_1.0mg/kg----[1] 19 💬
234GC1119----[1] 19 💬
235GC1123----[1] 19 💬
236Gelatine[1] Gelatin[2] Gelatin ,
Donkey glue 💬		--[1] 19 💬
237Gene Activated a-Galactosidase A----[1] 19 💬
238Gene Activated Human Glucocerebrosidase----[1] 19 💬
239Gene activated human glucocerebrosidase, velaglucerase alfa[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
240Gene-Activated Human Glucocerebrosidase----[1] 19 💬
241Gene-Activated Human Glucocerebrosidase 200U/vial----[1] 19 💬
242Gene-Activated Human Glucocerebrosidase 400U/vial----[1] 19 💬
243Genetic analyses----[1] 19 💬
244Genetic HMI-203----[1] 19 💬
245GENISTEIN[1] Genistein[1] Genistein 💬--[2] 19, 299 💬
246Genistein aglycone[1] Genistein[1] Genistein 💬--[1] 19 💬
247Genz-112638----[1] 19 💬
248GENZ-682452[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67 💬
249Genz-682452-AA[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
250Genz-682452-AU[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67 💬
251GLA----[2] 13, 19 💬
252GLA gene----[1] 19 💬
253GLA genetic sequencing.----[1] 19 💬
254GLUCOCEREBROSIDASE----[1] 19 💬
255GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO----[1] 19 💬
256GNR-055----[1] 19 💬
257GNR-055 1.0-2.0-3.0 mg/kg----[1] 19 💬
258GNR-055 2.0 mg/kg----[1] 19 💬
259GNR-055 3.0 mg/kg----[1] 19 💬
260GR181413----[1] 19 💬
261GR181413A----[1] 19 💬
262GR181413A/AT1001 capsule----[1] 19 💬
263GR181413A/AT1001 solution----[1] 19 💬
264Growth hormone[1] Somatotropin---[14] 2, 13, 19, 46, 78, 96, 107, 187, 191, 193, 195, 265, 276, 299 💬
265GSK2788723----[1] 19 💬
266GZ/SAR402671----[2] 19, 67 💬
267GZ385660----[1] 19 💬
268GZ402665----[1] 19 💬
269GZ402671----[2] 19, 67 💬
270GZ402671 / SAR402671----[1] 19 💬
271Hematopoetic Stem Cell Transplantation----[5] 19, 65, 120, 160, 326 💬
272Hematopoietic stem cell infusion----[2] 19, 20 💬
273Hematopoietic Stem Cell Transplantation----[12] 11, 13, 14, 19, 20, 35, 46, 49, 51, 96, 234, 285 💬
274Heparán N sulfatasa humana recombinante----[1] 19 💬
275Heparán N sulfatasa humana recombinante (rhHNS)----[1] 19 💬
276HGT-1110----[1] 19 💬
277HGT-1111----[1] 19 💬
278HGT-1410----[1] 19 💬
279HGT-2310----[1] 19 💬
280HIDS DONOR----[1] 19 💬
281HuCNS-SC----[1] 19 💬
282Human Glucocerebrosidase (prGCD)[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
283Human glucocerebrosidase cDNA----[1] 19 💬
284Human glucocerebrosidase gene into autologous peripheral blood stem cells----[1] 19 💬
285Human Placental Derived Stem Cell----[4] 19, 20, 60, 284 💬
286Hunterase[1] Idursulfase beta[1] Idursulfase beta 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
287Hydroxypropyl-beta-cyclodextrin[1] Betadex[1] Betadex 💬--[1] 19 💬
288Hydroxypropyl-ß-cyclodextrin----[1] 19 💬
289Hydroxyurea[1] Hydroxyurea[1] Hydroxyurea 💬[1] RRM2 💬[7] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Nucleotide metabolism, Purine metabolism, Pyrimidine metabolism, p53 signaling pathway 💬[8] 3, 13, 19, 20, 65, 85, 86, 284 💬
290I2S----[1] 19 💬
291I2S-IT----[1] 19 💬
292IB1001----[2] 19, 65 💬
293Iduronate 2-sulfatase[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
294Iduronate-2-sulfatase enzyme replacement therapy----[1] 19 💬
295IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR----[1] 19 💬
296IDURSULFASA[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
297IDURSULFASE[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
298Idursulfase (ELAPRASE)[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
299Idursulfase (I2S)[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
300Idursulfase (I2S)-IT[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
301Idursulfase beta[2] Idursulfase,
Idursulfase beta		[2] Idursulfase ,
Idursulfase beta 💬		[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
302Idursulfase IT (1 mg)[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
303Idursulfase IT (10 mg)[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
304Idursulfase IT (30 mg)[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
305Idursulfase(12S)-IT[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
306Idursulfase(I2S)-IT[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
307Idursulfase-IT[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
308IGF2----[1] 19 💬
309IMD----[5] 19, 20, 125, 234, 326 💬
310IMD Preparative Regimen----[5] 19, 20, 125, 234, 326 💬
311IMIGLUCERASA[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
312IMIGLUCERASE[1] Imiglucerase[1] Imiglucerase 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
313Imiglucerasi----[1] 19 💬
314Imiglucérase (drug) pharmacokinetics----[1] 19 💬
315Inactive Reminder Capsules----[1] 19 💬
316Inactive Reminder Product----[1] 19 💬
317INN Not available----[2] 19, 46 💬
318INNOZIDE----[1] 19 💬
319Intracerebral administration of AAVrh.10cuARSA----[1] 19 💬
320Intrathecal recombinant human alpha iduronidase----[1] 19 💬
321Iohexol[1] Iohexol[1] Iohexol 💬--[2] 19, 67 💬
322IRT Laronidase[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
323Isofagamine tartrate----[1] 19 💬
324ISU302----[1] 19 💬
325IV migalastat HCl[1] Migalastat[1] Migalastat 💬--[1] 19 💬
326IVA 336----[1] 19 💬
327JR-141----[1] 19 💬
328JR-141 or Idursulfase[1] Idursulfase[1] Idursulfase 💬[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
329JR-171 (lepunafusp alfa)----[1] 19 💬
330KANUMA[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
331LAMAZYM[1] Velmanase alfa[1] Velmanase alfa 💬[1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19 💬
332LARONIDASE[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[2] 19, 256 💬
333Laronidase ERT[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
334Laronidase therapy and a stem cell transplant[1] Laronidase[1] Laronidase 💬[1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
335Ledipasvir[1] Ledipasvir[1] Ledipasvir 💬--[1] 19 💬
336Ledipasvir/Sofosbuvir[2] Ledipasvir,
Sofosbuvir		[2] Sofosbuvir ,
Ledipasvir 💬		--[1] 19 💬
337Lenograstim[1] Lenograstim[1] Lenograstim 💬[1] CSF3R 💬[5] Cytokine-cytokine receptor interaction, Hematopoietic cell lineage, JAK-STAT signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer 💬[7] 2, 13, 16, 18, 19, 57, 65 💬
338Lentivirus Alpha-gal A transduced stem cells----[1] 19 💬
339Lentivirus-mediated delivery of ARSA to the CNS.----[1] 19 💬
340Leucine[1] Leucine[1] Leucine 💬--[6] 6, 19, 65, 283, 284, 299 💬
341Leucovorin[1] Leucovorin[2] Folinic acid ,
Folinic acid calcium salt pentahydrate 💬		--[2] 19, 46 💬
342Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA)----[1] 19 💬
343Lisinopril[1] Lisinopril[2] Lisinopril ,
Lisinopril 💬		[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[7] 19, 46, 49, 66, 67, 113, 222 💬
344Lopace----[1] 19 💬
345Losartan[1] Losartan[1] Losartan 💬[1] AGTR1 💬[16] 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] 17, 19, 36, 58, 66, 67, 85, 96, 97, 98, 113, 164, 167, 179, 215, 222, 299 💬
346Losartan and other angiotensin receptor blockers[2] Angiotensin II,
Losartan		[3] Angiotensin II ,
Angiotensin II ,
Losartan 💬		[2] AGTR1,
AGTR2 💬		[16] 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] 19 💬
347Lucerastat[1] Lucerastat[1] Lucerastat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
348LY3884961[1] LY3884961---[1] 19 💬
349Lymphocyte gene therapy----[1] 19 💬
350LYS----[1] 19 💬
351LYS-GM101----[1] 19 💬
352LYS-SAF302----[1] 19 💬
353Lysodase----[1] 19 💬
354Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬--[1] 19 💬
355Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬--[1] 19 💬
356MabThera[1] Rituximab[1] Rituximab 💬[1] MS4A1 💬[1] Hematopoietic cell lineage 💬[22] 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 ML[1] Rituximab[1] Rituximab 💬[1] MS4A1 💬[1] Hematopoietic cell lineage 💬[2] 19, 65 💬
358Medication to suppress the immune system----[1] 19 💬
359Melphalan[1] Melphalan[1] Melphalan 💬--[15] 11, 13, 14, 19, 20, 28, 60, 62, 65, 96, 164, 234, 284, 285, 299 💬
360Mepsevii[1] Vestronidase alfa[1] Vestronidase alfa 💬[1] GUSB 💬[8] Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬[1] 19 💬
361Mercaptamina[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
362Mercaptamine[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
363MERCAPTAMINE BITARTRATE[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
364MERCAPTAMINE HYDROCHLORIDE[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
365Mercaptamine, mercamine, 2-aminoethanethiol[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
366Metazym----[1] 19 💬
367Methotrexate[1] Methotrexate[1] Methotrexate 💬[2] DHFR,
DHFR2 💬		[5] Antifolate resistance, Biosynthesis of cofactors, Folate biosynthesis, Metabolic pathways, One carbon pool by folate 💬[38] 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 💬
368Methylprednisolone[1] Methylprednisolone[1] Methylprednisolone 💬[1] NR3C1 💬[1] Neuroactive ligand-receptor interaction 💬[46] 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 💬
369MIGALASTAT[1] Migalastat[1] Migalastat 💬--[1] 19 💬
370Migalastat HCl[1] Migalastat[1] Migalastat 💬--[1] 19 💬
371Migalastat HCl 150 mg[1] Migalastat[1] Migalastat 💬--[1] 19 💬
372MIGALASTAT HYDROCHLORIDE[1] Migalastat[1] Migalastat 💬--[1] 19 💬
373Migalastat hydrocloride[1] Migalastat[1] Migalastat 💬--[1] 19 💬
374MIGLUSTAT[1] Miglustat[1] Miglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 19, 256, 299 💬
375MIGLUSTAT (ZAVESCA)[1] Miglustat[1] Miglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
376Moss-aGal (recombinant human alpha-galactosidase A produced in moss)[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
377Mozobil[1] Plerixafor[1] Plerixafor 💬[1] CXCR4 💬[14] 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] 19, 65, 285 💬
378MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F[1] Plerixafor[1] Plerixafor 💬[1] CXCR4 💬[14] 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] 19 💬
379Mucoangin----[1] 19 💬
380Mycophenolate[1] Mycophenolic acid[1] Mycophenolic acid 💬[2] IMPDH1,
IMPDH2 💬		[4] Drug metabolism - other enzymes, Metabolic pathways, Nucleotide metabolism, Purine metabolism 💬[36] 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 mofetil[2] Mycophenolate mofetil,
Mycophenolic acid		[2] Mycophenolate mofetil ,
Mycophenolic acid 💬		[2] IMPDH1,
IMPDH2 💬		[4] Drug metabolism - other enzymes, Metabolic pathways, Nucleotide metabolism, Purine metabolism 💬[34] 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] 19, 20 💬
383MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML----[2] 19, 65 💬
384N acetyl cysteine[1] Cysteine---[1] 19 💬
385N Acetyl L Leucine[1] Leucine[1] Leucine 💬--[1] 19 💬
386N-Acetyl-L Leucine[1] Leucine[1] Leucine 💬--[1] 19 💬
387N-Acetyl-L-Leucine[2] L-Acetylleucine,
Leucine		[1] Leucine 💬--[2] 19, 65 💬
388N-Acetylcysteine[1] Acetylcysteine[1] Acetylcysteine 💬--[13] 6, 13, 19, 20, 21, 49, 58, 85, 90, 111, 164, 299, 337 💬
389N-acetylgalactosamine 4-sulfatase----[1] 19 💬
390Naglazyme[1] Galsulfase[1] Galsulfase 💬[1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
391Naglazyme (galsulfase)[1] Galsulfase[1] Galsulfase 💬[1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
392Naglazyme®[1] Galsulfase[1] Galsulfase 💬[1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
393NGN-101----[1] 19 💬
394Non applicable----[2] 10, 19 💬
395Odiparcil[1] Odiparcil---[1] 19 💬
396OGT 918----[2] 19, 299 💬
397OGT918----[1] 19 💬
398OGT923----[1] 19 💬
399OLENASUFLIGENE RELDUPARVOVEC[1] Olenasufligene relduparvovec---[1] 19 💬
400OLIPUDASE ALFA[1] Olipudase alfa[1] Olipudase alfa 💬[1] SMPD1 💬[5] Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19 💬
401Olipudase alfa (GZ402665)[1] Olipudase alfa[1] Olipudase alfa 💬[1] SMPD1 💬[5] Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19 💬
402Olipudase alfa (rhASM)[1] Olipudase alfa[1] Olipudase alfa 💬[1] SMPD1 💬[5] Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19 💬
403OMNIPAQUE[1] Iohexol[1] Iohexol 💬--[2] 19, 222 💬
404Oral migalastat HCl[1] Migalastat[1] Migalastat 💬--[1] 19 💬
405Osteopetrosis Haploidentical Only Preparative Regimen----[5] 19, 20, 125, 234, 326 💬
406Osteopetrosis Only Preparative Regimen----[5] 19, 20, 125, 234, 326 💬
407OTL-200----[1] 19 💬
408OTL-200 Dispersion for Infusion----[1] 19 💬
409OTL-200 Gene Therapy----[1] 19 💬
410Pabinafusp alfa[1] Pabinafusp alfa[1] Pabinafusp alfa 💬[2] IDS,
TFRC 💬		[8] Endocytosis, Ferroptosis, Glycosaminoglycan degradation, HIF-1 signaling pathway, Hematopoietic cell lineage, Lysosome, Metabolic pathways, Phagosome 💬[1] 19 💬
411PARACETAMOLO----[9] 2, 11, 14, 19, 28, 53, 61, 63, 300 💬
412PARACETAMOLO DOC GENERICI - 1000 MG COMPRESSA 16 COMPRESSE----[1] 19 💬
413Paricalcitol[1] Paricalcitol[1] Paricalcitol 💬[1] VDR 💬[5] Chemical carcinogenesis - receptor activation, Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis 💬[5] 19, 66, 218, 238, 298 💬
414PBGM01----[1] 19 💬
415PBKR03----[1] 19 💬
416PEGUNIGALSIDASE ALFA[1] Pegunigalsidase alfa[1] Pegunigalsidase alfa 💬--[1] 19 💬
417Pegunigalsidase alpha----[1] 19 💬
418Pending----[6] 13, 19, 96, 97, 113, 302 💬
419Pentosan Polysulfat SP 54® injection solution----[1] 19 💬
420Pentosan polysulfate[1] Pentosan polysulfate---[3] 19, 26, 226 💬
421PENTOSAN POLYSULFATE SODIUM[1] Pentosan polysulfate---[1] 19 💬
422Pentosan Polysulphate SP54----[1] 19 💬
423Pentosan Polysulphate SP54®----[1] 19 💬
424Perdix----[1] 19 💬
425Plant cell expressed recombinant glucocerebrosidase (prGCD)[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
426Plant Cell Recombinant Glucocerebrosidase----[1] 19 💬
427Plasma Lyso-GB3----[1] 19 💬
428PLX-200----[1] 19 💬
429Prednisone[1] Prednisone[1] Prednisone 💬[1] NR3C1 💬[1] Neuroactive ligand-receptor interaction 💬[45] 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/ALU[1] Prednisone[1] Prednisone 💬[1] NR3C1 💬[1] Neuroactive ligand-receptor interaction 💬[1] 19 💬
431Prevenar13----[2] 19, 265 💬
432PrGCD[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
433PROCYSBI[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
434PRX-102----[1] 19 💬
435PRX-102 (pegunigalsidase alfa)[1] Pegunigalsidase alfa[1] Pegunigalsidase alfa 💬--[1] 19 💬
436PRX-102 1 mg/kg every 2 weeks----[1] 19 💬
437PRX-102 2 mg/kg every 4 weeks----[1] 19 💬
438PRX-112----[1] 19 💬
439PRX102----[1] 19 💬
440PTC124----[4] 19, 113, 246, 299 💬
441Purified form of the lysosomal enzyme iduronate-2-sulfatase----[1] 19 💬
442PYRIMETHAMINE[1] Pyrimethamine[1] Pyrimethamine 💬--[4] 2, 4, 19, 65 💬
443RAAV 2/6 hIDS DONOR----[1] 19 💬
444RAAV2/5-hNAGLU----[1] 19 💬
445RAAV2/6 Left ZFN Vector----[1] 19 💬
446RAAV2/6 Right ZFN Vector----[1] 19 💬
447RAAV9.CMV.hNAGLU----[1] 19 💬
448Rebisufligene etisparvovec[1] Rebisufligene etisparvovec---[1] 19 💬
449Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A----[1] 19 💬
450Recombinant alpha-galactosidase A[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
451Recombinant human acid sphingomyelinase----[1] 19 💬
452RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)----[1] 19 💬
453Recombinant human alpha-galactosidase----[1] 19 💬
454Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL----[1] 19 💬
455Recombinant human alpha-mannosidase----[1] 19 💬
456Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)----[1] 19 💬
457RECOMBINANT HUMAN ARYLSULFATASE A----[1] 19 💬
458Recombinant Human Arylsulfatase A (rhASA)----[1] 19 💬
459Recombinant Human Arylsulfatase A (rhASA,----[1] 19 💬
460Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase----[1] 19 💬
461RECOMBINANT HUMAN BETA GLUCURONIDASE----[1] 19 💬
462Recombinant human beta-glucuronidase[1] Vestronidase alfa[1] Vestronidase alfa 💬[1] GUSB 💬[8] Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬[1] 19 💬
463Recombinant human beta-glucuronidase (rhGUS)[1] Vestronidase alfa[1] Vestronidase alfa 💬[1] GUSB 💬[8] Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬[1] 19 💬
464Recombinant human betaglucuronidase----[1] 19 💬
465Recombinant human derived macrophage-targeted beta-Glucocerebrosidase----[1] 19 💬
466Recombinant human derived macrophage-targeted ß-Glucocerebrosidase----[1] 19 💬
467Recombinant human glucerebrosidase----[1] 19 💬
468Recombinant human glucocerebrosidase----[1] 19 💬
469Recombinant Human Glucocerebrosidase (prGCD)[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
470Recombinant human heparan N-sulfatase----[1] 19 💬
471Recombinant human heparan N-sulfatase (rhHNS)----[1] 19 💬
472Recombinant human heparan N-sulfatase [rhHNS]----[1] 19 💬
473Recombinant human heparan Nsulfatase (rhHNS)----[1] 19 💬
474Recombinant human lysosomal acid lipase----[1] 19 💬
475Recombinant human lysosomal acid lipase (rhLAL)----[1] 19 💬
476Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬--[1] 19 💬
477Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬--[1] 19 💬
478Recombinant human lysosomal alpha-mannosidase----[1] 19 💬
479Recombinant human N-acetylgalactosamine 4-sulfatase----[1] 19 💬
480Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110----[1] 19 💬
481Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance----[1] 19 💬
482Recombinant human N-acetylgalactosamine-6-sulfatase[1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
483Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)[1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
484Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110[1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
485Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance[1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
486Recombinant human Nacetylgalactosamine-----[1] 19 💬
487Recombinant human tripeptidyl peptidase-1----[1] 19 💬
488RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)----[1] 19 💬
489Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene----[1] 19 💬
490Reduced Intensity Conditioning----[2] 19, 65 💬
491Reminder capsules----[1] 19 💬
492Reminder Product----[1] 19 💬
493REPLAGAL[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
494Replagal (Agalsidase Alfa)[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
495Replagal agalsidase alfa[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
496REPLAGAL ®[1] Agalsidase alfa[1] Agalsidase alfa 💬[1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
497RGX-111----[1] 19 💬
498RGX-121----[1] 19 💬
499Rh Growth Hormone[1] Somatotropin---[1] 19 💬
500RhARSA----[1] 19 💬
501RhASA----[1] 19 💬
502RhASA - Dose Level 1----[1] 19 💬
503RhASA - Dose Level 2----[1] 19 💬
504RhASA - Dose Level 3----[1] 19 💬
505RhASM----[1] 19 💬
506RhGALNS[1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
507RHGUS----[1] 19 💬
508RhHNS-10 mg----[1] 19 💬
509RhHNS-45 mg----[1] 19 💬
510RhHNS-90 mg----[1] 19 💬
511RhIDU (recombinant human-Alpha-L-Iduronidase)----[1] 19 💬
512RhLAMAN[1] Velmanase alfa[1] Velmanase alfa 💬[1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19 💬
513RhNAGLU----[1] 19 💬
514RhNAGLU radiolabelled----[1] 19 💬
515RHNAGLU-IGF2----[1] 19 💬
516Rimiducid[1] Rimiducid[1] Rimiducid 💬[1] CASP9 💬[35] 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] 19, 60, 65, 284, 285, 326 💬
517RITUXIMAB[1] Rituximab[1] Rituximab 💬[1] MS4A1 💬[1] Hematopoietic cell lineage 💬[48] 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] 19, 60, 285, 326 💬
519RP 103----[2] 8, 19 💬
520RP103----[2] 8, 19 💬
521RVX000222----[1] 19 💬
522SAF-301----[1] 19 💬
523SAR402671, GZ402671 o GZ/SAR402671----[2] 19, 67 💬
524SAR402671, GZ402671 or GZ / SAR402671----[1] 19 💬
525SAR402671, GZ402671 or GZ/SAR402671----[3] 6, 19, 67 💬
526SB-318----[1] 19 💬
527SB-47171----[1] 19 💬
528SB-47898----[1] 19 💬
529SB-913----[1] 19 💬
530SB-A6P-HRL Donor Vector----[1] 19 💬
531SB-FIX----[1] 19 💬
532SB-IDUA----[1] 19 💬
533SBC-102[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
534SBC-102, recombinant human lysosomal acid lipase[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
535SBC-103----[1] 19 💬
536ScAAV9.CB.CLN6----[1] 19 💬
537ScAAV9.U1A.hSGSH----[1] 19 💬
538ScAAV9.U1A.SGSH----[1] 19 💬
539Sebelipasa Alfa[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
540Sebelipase Alfa[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
541Sebelipase alfa (SBC-102)[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
542Sebelipase alfa 0.35 mg/kg[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
543Sebelipase alfa 1 mg/kg[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
544Sebelipase alfa 3 mg/kg[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
545SHP611----[1] 19 💬
546SHP675----[1] 19 💬
547Sirolimus[1] Sirolimus[1] Sirolimus 💬[1] MTOR 💬[51] 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] 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] 19 💬
549Sofosbuvir[1] Sofosbuvir[1] Sofosbuvir 💬--[1] 19 💬
550Somatropin[1] Somatotropin---[19] 2, 3, 19, 46, 65, 74, 75, 78, 96, 107, 113, 187, 193, 195, 238, 265, 274, 276, 299 💬
551Somatropin (DNA origin)[1] Somatotropin---[1] 19 💬
552Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride[1] Cysteamine[1] Cysteamine 💬--[1] 19 💬
553ST-920----[1] 19 💬
554Stem Cell Transplantation----[10] 13, 19, 20, 46, 65, 90, 125, 164, 234, 326 💬
555SUB195712----[1] 19 💬
556TALIGLUCERASE ALFA[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
557Taliglucerase alfa - Recombinant human glucocerebrosidase[1] Taliglucerase alfa[1] Taliglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
558TBD----[5] 19, 86, 90, 276, 299 💬
559Thiotepa[1] Thiotepa[1] Thiotepa 💬--[12] 13, 19, 20, 28, 51, 60, 65, 85, 96, 164, 284, 285 💬
560Thiotepa--escalated dose[1] Thiotepa[1] Thiotepa 💬--[4] 19, 20, 65, 284 💬
561Thiotepa--single daily dose[1] Thiotepa[1] Thiotepa 💬--[4] 19, 20, 65, 284 💬
562Thyroxine[1] Levothyroxine[1] Levothyroxine 💬[2] THRA,
THRB 💬		[2] Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬[1] 19 💬
563Tralesinidase alfa----[1] 19 💬
564Transduced CD34+ hematopoietic stem cell----[2] 19, 20 💬
565Trappsol Cyclo----[1] 19 💬
566TREOSULFAN[1] Treosulfan[1] Treosulfan 💬--[6] 13, 19, 62, 65, 283, 286 💬
567Triapin----[1] 19 💬
568Triapin& triapin Mite----[1] 19 💬
569TSHA-101----[1] 19 💬
570Unrelated Umbilical Cord Blood Transplant----[1] 19 💬
571UPLYSO----[1] 19 💬
572USAN: sebelipasa alfa[1] Sebelipase alfa[1] Sebelipase alfa 💬[1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19 💬
573USAN: sebelipase alpha----[1] 19 💬
574UX003----[1] 19 💬
575Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana----[1] 19 💬
576VELAGLUCERASE ALFA[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
577Velaglucerasi alfa----[1] 19 💬
578VELMANASE ALFA[1] Velmanase alfa[1] Velmanase alfa 💬[1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19 💬
579Velmanase Alfa (e.g. Lamazym)[1] Velmanase alfa[1] Velmanase alfa 💬[1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19 💬
580Venglustat[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67 💬
581Venglustat (GZ402671)[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
582Venglustat GZ402671[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[2] 19, 67 💬
583Venglustat malate[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67 💬
584Venglustat malato[1] Venglustat[1] Venglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[2] 19, 67 💬
585Vestronidase alfa[1] Vestronidase alfa[1] Vestronidase alfa 💬[1] GUSB 💬[8] Ascorbate and aldarate metabolism, Biosynthesis of cofactors, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome, Metabolic pathways, Pentose and glucuronate interconversions, Porphyrin metabolism 💬[1] 19 💬
586Vimizim® (elosulfase alfa)[1] Elosulfase alfa[1] Elosulfase alfa 💬[1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19 💬
587Vitamin D[2] Cholecalciferol,
Vitamin D		[1] Cholecalciferol 💬[1] VDR 💬[5] Chemical carcinogenesis - receptor activation, Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis 💬[20] 13, 19, 41, 43, 44, 46, 49, 50, 68, 93, 95, 96, 97, 179, 222, 228, 235, 274, 298, 299 💬
588VPRIV[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
589VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
590VPRIV 400 Units powder for solution of infusion[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
591VPRIV ®,[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
592VPRIV®[1] Velaglucerase alfa[1] Velaglucerase alfa 💬[1] GBA1 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
593VTS-270----[1] 19 💬
594Warfarin[1] Warfarin[1] Warfarin 💬[2] NQO1,
VKORC1 💬		[7] 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] 6, 8, 13, 19, 46, 49, 66, 85, 86, 88, 91 💬
595ZAVESCA[1] Miglustat[1] Miglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 19, 256, 299 💬
596Zavesca (Miglustat)[1] Miglustat[1] Miglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
597ZAVESCA 84CPS 100MG[1] Miglustat[1] Miglustat 💬[1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19 💬
598Zestoretic 10,20[1] Lisinopril[2] Lisinopril ,
Lisinopril 💬		[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[1] 19 💬
599ZESTORIC----[1] 19 💬
600Zestril[1] Lisinopril[2] Lisinopril ,
Lisinopril 💬		[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[3] 19, 67, 222 💬