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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] D10359 --[1] 19
41-deoxygalactonojirimycin hydrochloride[1] Migalastat[1] D10359 --[1] 19
512 lead electrocardiogram----[1] 19
612S-IT----[1] 19
715 O Water[1] Water[1] D00001 --[1] 19
82(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID----[2] 19, 65
92-aminoethanethiol[1] Cysteamine[2] D03634 ,
D03635 		--[2] 19, 299
102-hour Holter Monitor[1] Methamidophos---[1] 19
112-HYDROXYPROPYL-BETA-CYCLODEXTRIN[2] Betadex,
Hydroxypropyl betadex		[1] D02401 --[1] 19
124D-310----[1] 19
13500 0500 0----[1] 19
1489 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)----[1] 19
1589Zr-SBC-103----[1] 19
16[14C] AT1001----[1] 19
17A (migalastat)[1] Migalastat[1] D10359 --[1] 19
18A16AB04----[1] 19
19A16AX06----[1] 19
20AAV2/8.TBG.hARSB----[1] 19
21AAV2CUhCLN2 (3x10^12 particle units)----[1] 19
22AAV9-CAG-coh-SGSH----[1] 19
23AAV9-GLB1----[1] 19
24AAVrh.10-hMPSIIIA----[1] 19
25AAVrh.10cuARSA----[1] 19
26AAVrh.10CUCLN2----[1] 19
27AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies----[1] 19
28AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies----[1] 19
29Abatacept[1] Abatacept[1] D03203 [2] CD80,
CD86 💬		[12] Allograft rejection, Autoimmune thyroid disease, Cell adhesion molecules, Graft-versus-host disease, Intestinal immune network for IgA production, Kaposi sarcoma-associated herpesvirus infection, Rheumatoid arthritis, Systemic lupus erythematosus, Toll-like receptor signaling pathway, Transcriptional misregulation in cancer, Type I diabetes mellitus, Viral myocarditis 💬[26] 11, 13, 19, 41, 44, 46, 49, 50, 51, 53, 55, 60, 65, 84, 93, 95, 96, 97, 107, 160, 164, 222, 271, 284, 285, 300
30Abcertin----[1] 19
31ABO-101----[1] 19
32ABO-102----[1] 19
33Accupro----[1] 19
34Accuretic[1] Quinapril[2] D00459 ,
D03752 		[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[1] 19
35Acetylcysteine[1] Acetylcysteine[1] D00221 --[20] 6, 13, 19, 20, 34, 49, 51, 53, 58, 85, 90, 94, 111, 164, 193, 233, 296, 298, 299, 337
36ACT-434964----[1] 19
37Adalimumab[1] Adalimumab[1] D02597 [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 💬[18] 19, 37, 40, 41, 46, 56, 84, 96, 97, 107, 151, 160, 164, 222, 226, 269, 270, 271
38Adalimumab Injection [Humira][1] Adalimumab[1] D02597 [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 💬[1] 19
39ADENO-ASSOCIATED VIRAL (AAV) SEROTYPE 8 (AAV2/8) VECTOR WITH LIVER-SPECIFIC THYROXINE-BINDING GLOBULIN (TBG) PROMOTER, DRIVING THE EXPRESSION OF THE HUMAN ARSB GENE[1] Levothyroxine[2] D01010 ,
D08125 		[2] THRA,
THRB 💬		[2] Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬[1] 19
40Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene----[1] 19
41Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-betagal)----[1] 19
42Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal)----[1] 19
43Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
44Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
45Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector----[1] 19
46Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector----[1] 19
47Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector----[1] 19
48Adeno-associated virus serotype hu68 containing the human GALC gene----[1] 19
49Adeno-associated virus serotype rh.10 expressing beta-galactosidase[1] Tilactase[1] D03348 --[1] 19
50ADMINISTRATION----[1] 19
51Administration Kit----[1] 19
52Adrabetadex[1] Adrabetadex---[1] 19
53Afegostat[1] Afegostat[1] D09576 --[1] 19
54Afegostat tartrate[1] Afegostat[1] D09576 --[1] 19
55Agalsidase----[1] 19
56AGALSIDASE ALFA[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
57Agalsidase Alpha[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
58AGALSIDASE BETA[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
59Agalsidase beta (GZ419828)[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
60Agalsidase beta (recombinant form)[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
61AGALSIDASI ALFA----[1] 19
62AGALSIDASI BETA----[1] 19
63AGT-181----[1] 19
64AGT-181 (HIRMAb-IDUA)----[1] 19
65AGT-182----[1] 19
66Aldurazyme[1] Laronidase[1] D04670 [1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
67Aldurazyme (laronidase)[1] Laronidase[1] D04670 [1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[2] 19, 256
68Aldurazyme (Recombinant Human Alpha-L-Iduronidase)[1] Laronidase[1] D04670 [1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
69ALDURAZYME*INFUS 1FL 5ML 500U[1] Laronidase[1] D04670 [1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
70ALEMTUZUMAB[1] Alemtuzumab[1] D02802 [1] CD52 💬-[17] 13, 15, 19, 20, 36, 43, 44, 46, 51, 60, 63, 65, 85, 96, 164, 283, 284
71Alendronate[1] Alendronic acid[2] D00939 ,
D07119 		[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
72Alendronate sodium[1] Alendronic acid[2] D00939 ,
D07119 		[1] FDPS 💬[4] Human T-cell leukemia virus 1 infection, Influenza A, Metabolic pathways, Terpenoid backbone biosynthesis 💬[2] 19, 299
73Alfa-manosidadas humana recombinante----[1] 19
74Allogeneic stem cell transplantation----[5] 19, 20, 49, 62, 84
75Alpha-galactosidase A[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
76Ambroxol[1] Ambroxol[1] D07442 --[2] 6, 19
77Anakinra[1] Anakinra[1] D02934 [2] IL1R1,
IL1R2 💬		[15] Amoebiasis, Cytokine-cytokine receptor interaction, Fluid shear stress and atherosclerosis, Hematopoietic cell lineage, Human T-cell leukemia virus 1 infection, Human cytomegalovirus infection, Inflammatory mediator regulation of TRP channels, MAPK signaling pathway, NF-kappa B signaling pathway, Osteoclast differentiation, Pathogenic Escherichia coli infection, Prostate cancer, Shigellosis, Th17 cell differentiation, Transcriptional misregulation in cancer 💬[17] 2, 13, 15, 19, 28, 37, 41, 46, 49, 50, 56, 84, 86, 97, 106, 107, 299
78Angiotensin[1] Angiotensin II[2] D00150 ,
D02014 		[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] D11374 --[3] 2, 15, 19
80Arimoclomol (hard gelatine capsule)[2] Arimoclomol,
Gelatin		[3] D00115 ,
D06892 ,
D11374 		--[1] 19
81Arimoclomol (HPMC capsule)[1] Arimoclomol[1] D11374 --[1] 19
82Arimoclomol citrate[1] Arimoclomol[1] D11374 --[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] D09323 --[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 Plasmablasts----[1] 19
92AVR-RD-01----[1] 19
93AVR-RD-02----[1] 19
94AX----[1] 19
95AX 250----[1] 19
96AXO-AAV-GM2 High Dose----[1] 19
97AXO-AAV-GM2 Low Dose----[1] 19
98AXO-AAV-GM2 Middle Dose----[1] 19
99AXO-AAV-GM2 Starting Dose----[1] 19
100Azathioprine[1] Azathioprine[2] D00238 ,
D03033 		--[21] 6, 11, 13, 19, 35, 42, 43, 44, 45, 46, 49, 63, 65, 66, 85, 93, 94, 95, 96, 97, 162
101Azathioprine (AZA)[1] Azathioprine[2] D00238 ,
D03033 		--[3] 13, 19, 97
102B (migalastat)[1] Migalastat[1] D10359 --[1] 19
103Barium[1] Barium---[3] 2, 19, 98
104Behavioral: Neurocognitive testing----[1] 19
105Behavioral: PICC line placement----[1] 19
106Beta-cyclodextrin[1] Betadex[1] D02401 --[1] 19
107Beta-Galactosidase[1] Tilactase[1] D03348 --[1] 19
108Beta-Mercaptoethylamine[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
109Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
110BHP001----[1] 19
111Biological assays----[1] 19
112Biopsy----[4] 19, 51, 78, 193
113Blood draw----[5] 6, 19, 65, 231, 299
114Blood sampling----[6] 13, 17, 19, 46, 51, 96
115BM110----[1] 19
116BMN 110----[1] 19
117BMN 110 - Every Other Week----[1] 19
118BMN 110 - Weekly----[1] 19
119BMN 110 Every Other Week----[1] 19
120BMN 110 Weekly----[1] 19
121BMN 190----[1] 19
122BMN 250----[1] 19
123BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)----[1] 19
124Brain MRI/MRS/fMRI----[1] 19
125Brineura[1] Cerliponase alfa[1] D10813 [1] TPP1 💬[1] Lysosome 💬[1] 19
126BUSILVEX[1] Busulfan[1] D00248 --[4] 19, 36, 60, 65
127BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI[1] Busulfan[1] D00248 --[2] 19, 65
128Busulfan[1] Busulfan[1] D00248 --[13] 19, 20, 28, 36, 49, 51, 60, 65, 164, 274, 284, 285, 326
129Busulfan, Cyclophosphamide, Antithymocyte Globulin[2] Busulfan,
Cyclophosphamide		[3] D00248 ,
D00287 ,
D07760 		--[2] 19, 20
130Busulfan, Cyclophosphamide, ATG[2] Busulfan,
Cyclophosphamide		[3] D00248 ,
D00287 ,
D07760 		--[1] 19
131BUSULFANO[1] Busulfan[1] D00248 --[1] 19
132C (migalastat)[1] Migalastat[1] D10359 --[1] 19
133Calcium[1] Calcium---[30] 3, 6, 13, 15, 19, 46, 49, 51, 53, 56, 68, 75, 79, 84, 93, 94, 95, 96, 97, 186, 214, 215, 222, 224, 235, 256, 265, 274, 298, 299
134Calcium carbonate[2] Calcium,
Carbonate ion		---[8] 13, 19, 46, 53, 75, 96, 235, 299
135CALD HR-D (High-Risk, Regimen C)----[5] 19, 20, 125, 234, 326
136CALD HR-D (High-Risk, Regimen D)----[5] 19, 20, 125, 234, 326
137CALD SR-A (Standard-Risk, Regimen A)----[5] 19, 20, 125, 234, 326
138CALD SR-B (Standard-Risk, Regimen B)----[5] 19, 20, 125, 234, 326
139Campath[1] Alemtuzumab[1] D02802 [1] CD52 💬-[12] 19, 20, 49, 51, 60, 62, 65, 164, 234, 283, 285, 326
140Campath-1H[1] Alemtuzumab[1] D02802 [1] CD52 💬-[6] 19, 20, 60, 234, 285, 326
141Cannabis[1] Medical Cannabis---[9] 2, 6, 13, 19, 46, 90, 96, 97, 271
142Cannabis sativa L., folium cum flore[1] Medical Cannabis---[1] 19
143Capsaicin[1] Capsaicin[1] D00250 [1] TRPV1 💬[2] Inflammatory mediator regulation of TRP channels, Neuroactive ligand-receptor interaction 💬[5] 2, 6, 19, 86, 226
144Carbonate[1] Carbonate ion---[12] 2, 13, 17, 18, 19, 46, 53, 75, 96, 127, 235, 299
145Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi----[1] 19
146Cerdelga[1] Eliglustat[1] D09893 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
147Ceredase[1] Alglucerase[1] D02810 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
148CEREDASE™[1] Alglucerase[1] D02810 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
149CEREZYME[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
150Cerezyme (imiglucerase for injection)[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
151Cerezyme 400 Units Powder for concentrate for solution for infusion[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
152Cerezyme®[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
153Cerezyme® / Imiglucerase[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
154Cerliponase Alfa[1] Cerliponase alfa[1] D10813 [1] TPP1 💬[1] Lysosome 💬[1] 19
155CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE----[1] 19
156CHOLECALCIFEROL[1] Cholecalciferol[1] D00188 [1] VDR 💬[5] Chemical carcinogenesis - receptor activation, Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis 💬[10] 13, 19, 34, 46, 49, 75, 96, 97, 298, 299
157Cholesterol[1] Cholesterol[1] D00040 --[6] 19, 90, 160, 259, 261, 310
158Clofarabine[1] Clofarabine[1] D03546 [3] POLA1,
POLA2,
RRM1 💬		[6] DNA replication, Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬[9] 19, 20, 40, 46, 49, 60, 96, 234, 326
159Contrast sensitivity measurement----[1] 19
160Coversyl[1] Perindopril[2] D00624 ,
D03753 		[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[2] 19, 167
161Coversyl PLUS----[1] 19
162CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE----[1] 19
163CsA----[8] 19, 49, 50, 53, 60, 63, 222, 228
164Cyclophosphamide[1] Cyclophosphamide[2] D00287 ,
D07760 		--[45] 11, 13, 14, 16, 19, 20, 26, 28, 35, 36, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 56, 60, 62, 65, 66, 84, 85, 93, 96, 162, 164, 172, 205, 222, 224, 256, 265, 274, 283, 284, 285, 288, 326, 331
165Cyclosporine[1] Cyclosporine[1] D00184 [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
166Cyclosporine A[1] Cyclosporine[1] D00184 [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
167Cyclosporine A (CsA)[1] Cyclosporine[1] D00184 [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
168Cystadane anhydrous----[1] 19
169CYSTADROPS[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
170CYSTADROPS 0.55% eye drops, solution[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
171CYSTAGON[1] Cysteamine[2] D03634 ,
D03635 		--[3] 8, 19, 299
172Cystagon 150mg[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
173Cystagon 50mg[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
174Cystagon®[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
175Cystagon® (Cysteamine Bitartrate)[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
176Cysteamine[1] Cysteamine[2] D03634 ,
D03635 		--[4] 8, 19, 21, 299
177Cysteamine (mercaptamine) viscous solution[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
178Cysteamine Bitartrate[1] Cysteamine[2] D03634 ,
D03635 		--[4] 8, 19, 21, 299
179Cysteamine bitartrate (INN: mercaptamine bitartrate[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
180Cysteamine bitartrate (INN: mercaptamine bitartrate )[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
181Cysteamine bitartrate (INN: mercaptamine bitartrate)[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
182Cysteamine Bitartrate Delayed-release Capsules[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
183Cysteamine Bitartrate Delayed-release Capsules (RP103)[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
184CYSTEAMINE HYDROCHLORIDE[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
185Cysteine[1] Cysteine[2] D00026 ,
D02326 		--[11] 6, 13, 19, 34, 84, 85, 88, 90, 254, 296, 310
186D (migalastat)[1] Migalastat[1] D10359 --[1] 19
187DEXA scan----[3] 19, 46, 96
188Diagnostic Test: Audiology assessmentwith ABR----[1] 19
189Diagnostic Test: Bone density scan (DEXA----[1] 19
190Diagnostic Test: Echocardiogram----[1] 19
191Diagnostic Test: Echocardiography at M24----[1] 19
192Diagnostic Test: Echocardiography at T0----[1] 19
193Diagnostic Test: Electrocardiogram (EKG)----[1] 19
194Diagnostic Test: Exercise test----[1] 19
195Diagnostic Test: Laboratory tests----[1] 19
196Diagnostic Test: Plasma a-Gal A activity----[1] 19
197DNL310----[1] 19
198DNL310 Drug Substance----[1] 19
199Dried blood spot (DBS) sampling----[1] 19
200DRX005B----[1] 19
201DUOC-01----[3] 13, 19, 20
202E (migalastat)[1] Migalastat[1] D10359 --[1] 19
203Echocardiogram----[2] 19, 215
204Elaprase[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
205Elaprase 2mg/ml[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
206Elaprase for intravenous (IV) infusion[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
207Elaprase_0.5mg/kg----[1] 19
208Elelyso[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
209Elelyso 60 units/kg[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
210Eliglustat[1] Eliglustat[1] D09893 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
211Eliglustat GZ385660[1] Eliglustat[1] D09893 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
212ELIGLUSTAT TARTRATE[1] Eliglustat[1] D09893 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
213Eliglustat Tartrate – Powder for Oral Suspension[1] Eliglustat[1] D09893 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
214Elosulfase alfa[1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
215Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM][1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
216ELX-02[1] ELX-02---[2] 19, 299
217Enalapril[1] Enalapril[2] D00621 ,
D07892 		[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
218Enalapril and other angiotensin converting enzyme inhibitors[2] Angiotensin II,
Enalapril		[4] D00150 ,
D00621 ,
D02014 ,
D07892 		[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
219Enriched Hematopoetic Stem Cell Infusion----[3] 13, 19, 284
220Enzyme replacement----[1] 19
221Enzyme replacement therapy----[1] 19
222Enzyme replacement therapy (Agalsidase alpha (Replagal®))[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
223Enzyme replacement therapy (ERT)----[1] 19
224ERT----[1] 19
225EU-sourced Cerezyme[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
226FABRAZYME[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
227Fabrazyme (agalsidase beta)[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
228FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
229Fabrazyme 35 mg[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
230Fabrazyme 5 mg[1] Agalsidase beta[1] D03228 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
231FBX-101----[1] 19
232FLT190----[1] 19
233Fludarabina[1] Fludarabine[2] D01907 ,
D07966 		[1] RRM1 💬[5] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬[2] 19, 65
234FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML[1] Fludarabine[2] D01907 ,
D07966 		[1] RRM1 💬[5] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬[1] 19
235FLUDARABINA FOSFATO[1] Fludarabine[2] D01907 ,
D07966 		[1] RRM1 💬[5] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism 💬[1] 19
236Fluorescein[1] Fluorescein[2] D01261 ,
D02024 		--[3] 19, 22, 97
237GA-GCB[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
238GA-GCB (velaglucerase alfa)[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
239GABAPENTIN[1] Gabapentin[1] D00332 [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
240Galafold[1] Migalastat[1] D10359 --[1] 19
241GALSULFASE[1] Galsulfase[1] D06565 [1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
242GC1111_0.5mg/kg----[1] 19
243GC1111_1.0mg/kg----[1] 19
244GC1119----[1] 19
245Gelatine[1] Gelatin[2] D00115 ,
D06892 		--[1] 19
246Gene Activated a-Galactosidase A----[1] 19
247Gene Activated Human Glucocerebrosidase----[1] 19
248Gene activated human glucocerebrosidase, velaglucerase alfa[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
249Gene-Activated Human Glucocerebrosidase----[1] 19
250Gene-Activated Human Glucocerebrosidase 200U/vial----[1] 19
251Gene-Activated Human Glucocerebrosidase 400U/vial----[1] 19
252Genetic HMI-203----[1] 19
253Genetic: GLA gene----[1] 19
254Genetic: LYS-GM101----[1] 19
255GENISTEIN[1] Genistein[1] D11680 --[2] 19, 299
256Genistein aglycone[1] Genistein[1] D11680 --[1] 19
257Genz-112638----[1] 19
258GENZ-682452[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67
259Genz-682452-AA[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
260Genz-682452-AU[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67
261GLA----[2] 13, 19
262GLA genetic sequencing.----[1] 19
263GLUCOCEREBROSIDASE----[1] 19
264GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO----[1] 19
265GNR-055----[1] 19
266GR181413----[1] 19
267GR181413A----[1] 19
268GR181413A/AT1001 capsule----[1] 19
269GR181413A/AT1001 solution----[1] 19
270Growth hormone[1] Somatotropin[1] D02691 [1] GHR 💬[5] Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬[13] 2, 13, 19, 46, 78, 96, 107, 187, 191, 193, 195, 265, 299
271GSK2788723----[1] 19
272GZ/SAR402671----[2] 19, 67
273GZ385660----[1] 19
274GZ402665----[1] 19
275GZ402671----[2] 19, 67
276GZ402671 / SAR402671----[1] 19
277Hematopoetic Stem Cell Transplantation----[5] 19, 65, 120, 160, 326
278Hematopoietic stem cell infusion----[2] 19, 20
279Hematopoietic Stem Cell Transplantation----[13] 11, 13, 14, 19, 20, 35, 46, 49, 51, 65, 96, 234, 285
280Heparán N sulfatasa humana recombinante----[1] 19
281Heparán N sulfatasa humana recombinante (rhHNS)----[1] 19
282HGT-1110----[1] 19
283HGT-1111----[1] 19
284HGT-1410----[1] 19
285HGT-2310----[1] 19
286HIDS DONOR----[1] 19
287HuCNS-SC----[1] 19
288Human Glucocerebrosidase (prGCD)[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
289Human Placental Derived Stem Cell----[4] 19, 20, 60, 284
290Hunterase[1] Idursulfase beta[1] D11025 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
291Hydroxypropyl-beta-cyclodextrin[1] Betadex[1] D02401 --[1] 19
292Hydroxypropyl-ß-cyclodextrin----[1] 19
293HYDROXYPROPYLBETADEX----[1] 19
294Hydroxyurea[1] Hydroxyurea[1] D00341 [1] RRM2 💬[6] Drug metabolism - other enzymes, Glutathione metabolism, Metabolic pathways, Purine metabolism, Pyrimidine metabolism, p53 signaling pathway 💬[8] 3, 13, 19, 20, 65, 85, 86, 284
295I2S----[1] 19
296I2S-IT----[1] 19
297IB1001----[2] 19, 65
298Iduronate 2-sulfatase[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
299Iduronate-2-sulfatase enzyme replacement therapy----[1] 19
300IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR----[1] 19
301IDURSULFASA[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
302IDURSULFASE[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
303Idursulfase (I2S)[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
304Idursulfase (I2S)-IT[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
305Idursulfase beta[2] Idursulfase,
Idursulfase beta		[2] D04499 ,
D11025 		[1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
306Idursulfase IT (1 mg)[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
307Idursulfase IT (10 mg)[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
308Idursulfase IT (30 mg)[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
309Idursulfase(12S)-IT[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
310Idursulfase(I2S)-IT[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
311Idursulfase-IT[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
312IGF2----[1] 19
313IMD----[5] 19, 20, 125, 234, 326
314IMD Preparative Regimen----[5] 19, 20, 125, 234, 326
315IMIGLUCERASA[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
316IMIGLUCERASE[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
317Imiglucerase GZ437843[1] Imiglucerase[1] D03020 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
318Imiglucerasi----[1] 19
319Imiglucérase (drug) pharmacokinetics----[1] 19
320Inactive Reminder Capsules----[1] 19
321Inactive Reminder Product----[1] 19
322INN Not available----[2] 19, 46
323INNOZIDE----[1] 19
324Intrathecal recombinant human alpha iduronidase----[1] 19
325Intraventricular Access Device----[1] 19
326Iohexol[1] Iohexol[1] D01817 --[2] 19, 67
327IRT Laronidase[1] Laronidase[1] D04670 [1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
328Isofagamine tartrate----[1] 19
329ISU302----[1] 19
330IV migalastat HCl[1] Migalastat[1] D10359 --[1] 19
331IVA 336----[1] 19
332JR-141----[1] 19
333JR-141 or Idursulfase[1] Idursulfase[1] D04499 [1] IDS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
334JR-171----[1] 19
335KANUMA[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
336LAMAZYM[1] Velmanase alfa[1] D11024 [1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19
337LARONIDASE[1] Laronidase[1] D04670 [1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[2] 19, 256
338Laronidase ERT[1] Laronidase[1] D04670 [1] IDUA 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
339Ledipasvir[1] Ledipasvir[1] D10442 --[1] 19
340Ledipasvir/Sofosbuvir[2] Ledipasvir,
Sofosbuvir		[2] D10366 ,
D10442 		--[1] 19
341Lenograstim[1] Lenograstim[1] D03247 [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
342Lentivirus Alpha-gal A transduced stem cells----[1] 19
343Lentivirus-mediated delivery of ARSA to the CNS.----[1] 19
344Leucine[1] Leucine[1] D00030 --[11] 5, 6, 7, 19, 65, 96, 97, 127, 283, 284, 299
345Leucovorin[1] Leucovorin[3] D01211 ,
D07986 ,
D07987 		--[2] 19, 46
346Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA)----[1] 19
347Lisinopril[1] Lisinopril[2] D00362 ,
D08131 		[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
348LITHIUM CARBONATE[2] Carbonate ion,
Lithium carbonate		[1] D00801 --[6] 2, 13, 17, 18, 19, 127
349Lopace----[1] 19
350Losartan[1] Losartan[2] D00357 ,
D08146 		[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 💬[18] 17, 19, 36, 46, 58, 66, 67, 85, 96, 97, 98, 113, 164, 167, 179, 215, 222, 299
351Losartan and other angiotensin receptor blockers[2] Angiotensin II,
Losartan		[4] D00150 ,
D00357 ,
D02014 ,
D08146 		[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
352Lucerastat[1] Lucerastat[1] D11439 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
353Lumbar Puncture----[6] 6, 13, 19, 96, 97, 127
354LYS----[1] 19
355LYS-GM101----[1] 19
356LYS-SAF302----[1] 19
357Lysodase----[1] 19
358Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] D00040 --[1] 19
359Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] D00040 --[1] 19
360MabThera[1] Rituximab[1] D02994 [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
361MABTHERA - 2 FIALE 100 MG 10 ML[1] Rituximab[1] D02994 [1] MS4A1 💬[1] Hematopoietic cell lineage 💬[2] 19, 65
362Magnetic Resonance Imaging----[7] 6, 13, 19, 22, 34, 205, 299
363Mechanical stimulation with Von Frey filaments----[1] 19
364Medication to suppress the immune system----[1] 19
365Melphalan[1] Melphalan[1] D00369 --[15] 11, 13, 14, 19, 20, 28, 60, 62, 65, 96, 164, 234, 284, 285, 299
366Mepsevii[1] Vestronidase alfa[1] D11004 [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
367Mercaptamina[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
368Mercaptamine[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
369MERCAPTAMINE BITARTRATE[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
370MERCAPTAMINE HYDROCHLORIDE[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
371Mercaptamine, mercamine, 2-aminoethanethiol[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
372Metazym----[1] 19
373Methotrexate[1] Methotrexate[2] D00142 ,
D02115 		[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
374Methylprednisolone[1] Methylprednisolone[6] D00407 ,
D00751 ,
D00979 ,
D05000 ,
D05001 ,
D05002 		[1] NR3C1 💬[1] Neuroactive ligand-receptor interaction 💬[48] 2, 6, 11, 13, 14, 18, 19, 25, 26, 35, 38, 39, 40, 41, 42, 43, 45, 46, 49, 50, 51, 60, 62, 63, 64, 65, 66, 83, 84, 86, 93, 95, 96, 97, 127, 145, 162, 164, 211, 220, 222, 224, 271, 283, 284, 285, 296, 300
375MIGALASTAT[1] Migalastat[1] D10359 --[1] 19
376Migalastat HCl[1] Migalastat[1] D10359 --[1] 19
377Migalastat HCl 150 mg[1] Migalastat[1] D10359 --[1] 19
378MIGALASTAT HYDROCHLORIDE[1] Migalastat[1] D10359 --[1] 19
379Migalastat hydrocloride[1] Migalastat[1] D10359 --[1] 19
380MIGLUSTAT[1] Miglustat[1] D05032 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 19, 256, 299
381MIGLUSTAT (ZAVESCA)[1] Miglustat[1] D05032 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
382MONITOR[1] Methamidophos---[20] 2, 11, 13, 19, 28, 46, 49, 58, 65, 70, 81, 85, 86, 98, 113, 118, 129, 298, 299, 300
383Moss-aGal (recombinant human alpha-galactosidase A produced in moss)[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
384Mozobil[1] Plerixafor[1] D08971 [1] CXCR4 💬[12] Axon guidance, Calcium signaling pathway, Chemokine signaling pathway, Cytokine-cytokine receptor interaction, Endocytosis, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Intestinal immune network for IgA production, Leukocyte transendothelial migration, Pathways in cancer, Regulation of actin cytoskeleton, Viral protein interaction with cytokine and cytokine receptor 💬[3] 19, 65, 285
385MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F[1] Plerixafor[1] D08971 [1] CXCR4 💬[12] Axon guidance, Calcium signaling pathway, Chemokine signaling pathway, Cytokine-cytokine receptor interaction, Endocytosis, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Intestinal immune network for IgA production, Leukocyte transendothelial migration, Pathways in cancer, Regulation of actin cytoskeleton, Viral protein interaction with cytokine and cytokine receptor 💬[1] 19
386MPS----[1] 19
387MRI with contrast agent injection----[1] 19
388MRI without contrast agent injection----[1] 19
389Mycophenolate[1] Mycophenolic acid[1] D05096 [2] IMPDH1,
IMPDH2 💬		[3] Drug metabolism - other enzymes, Metabolic pathways, 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
390Mycophenolate mofetil[2] Mycophenolate mofetil,
Mycophenolic acid		[4] D00752 ,
D05094 ,
D05095 ,
D05096 		[2] IMPDH1,
IMPDH2 💬		[3] Drug metabolism - other enzymes, Metabolic pathways, 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
391Mycophenylate mofetil----[2] 19, 20
392MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML----[2] 19, 65
393N acetyl cysteine[1] Cysteine[2] D00026 ,
D02326 		--[2] 6, 19
394N Acetyl L Leucine[1] Leucine[1] D00030 --[1] 19
395N-acetyl Cysteine[1] Cysteine[2] D00026 ,
D02326 		--[7] 13, 19, 34, 85, 88, 296, 310
396N-Acetyl-L Leucine[1] Leucine[1] D00030 --[1] 19
397N-Acetyl-L-Leucine[1] Leucine[1] D00030 --[2] 19, 65
398N-Acetylcysteine[1] Acetylcysteine[1] D00221 --[11] 6, 13, 19, 20, 49, 58, 85, 111, 164, 299, 337
399N-acetylgalactosamine 4-sulfatase----[1] 19
400Naglazyme[1] Galsulfase[1] D06565 [1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
401Naglazyme (galsulfase)[1] Galsulfase[1] D06565 [1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
402Naglazyme®[1] Galsulfase[1] D06565 [1] ARSB 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
403Non applicable----[2] 10, 19
404Odiparcil[1] Odiparcil---[1] 19
405OGT 918----[2] 19, 299
406OGT918----[1] 19
407OGT923----[1] 19
408OLENASUFLIGENE RELDUPARVOVEC[1] Olenasufligene relduparvovec---[1] 19
409Olipudase alfa[1] Olipudase alfa[1] D10820 [1] SMPD1 💬[5] Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19
410Olipudase alfa (rhASM)[1] Olipudase alfa[1] D10820 [1] SMPD1 💬[5] Lysosome, Metabolic pathways, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19
411OMNIPAQUE[1] Iohexol[1] D01817 --[2] 19, 222
412Ophthalmology exam----[1] 19
413Oral migalastat HCl[1] Migalastat[1] D10359 --[1] 19
414OSOME----[1] 19
415Osteopetrosis Haploidentical Only Preparative Regimen----[5] 19, 20, 125, 234, 326
416Osteopetrosis Only Preparative Regimen----[5] 19, 20, 125, 234, 326
417Other: Brief Pain Inventory questionnaire----[1] 19
418Other: Challenge agent: capsaicin[1] Capsaicin[1] D00250 [1] TRPV1 💬[2] Inflammatory mediator regulation of TRP channels, Neuroactive ligand-receptor interaction 💬[1] 19
419Other: Control----[9] 6, 19, 67, 70, 75, 85, 97, 162, 193
420Other: Cysteamine bitartrate[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
421Other: Dried blood spot test for MPS----[1] 19
422Other: Electroencephalogram (EEG) awake andextended overnight----[1] 19
423Other: Fluorescein angiography[1] Fluorescein[2] D01261 ,
D02024 		--[1] 19
424Other: General and Neurological examination----[1] 19
425Other: Ketogenic Diet----[2] 19, 67
426Other: MPS I----[1] 19
427Other: Neurology exam----[1] 19
428Other: No IT treatment----[1] 19
429Other: Palliative Care----[5] 19, 65, 120, 160, 326
430Other: Parts A/B: Sham Control----[1] 19
431Other: Potable water[1] Water[1] D00001 --[1] 19
432Other: Quality of Life questionnaire----[1] 19
433Other: Vital signs----[1] 19
434OTL-200----[1] 19
435OTL-200 Dispersion for Infusion----[1] 19
436Oxygen[1] Oxygen[1] D00003 --[20] 6, 13, 19, 46, 49, 70, 84, 85, 86, 88, 90, 96, 97, 168, 193, 226, 271, 294, 299, 330
437Oxygen flow at the optic nerve head measurement[1] Oxygen[1] D00003 --[1] 19
438Pabinafusp alfa[1] Pabinafusp alfa[1] D11812 [2] IDS,
TFRC 💬		[8] Endocytosis, Ferroptosis, Glycosaminoglycan degradation, HIF-1 signaling pathway, Hematopoietic cell lineage, Lysosome, Metabolic pathways, Phagosome 💬[1] 19
439Paricalcitol[1] Paricalcitol[1] D00930 [1] VDR 💬[5] Chemical carcinogenesis - receptor activation, Endocrine and other factor-regulated calcium reabsorption, Mineral absorption, Parathyroid hormone synthesis, secretion and action, Tuberculosis 💬[4] 19, 66, 218, 238
440Parts A/B: Adrabetadex[1] Adrabetadex---[1] 19
441PBGM01----[1] 19
442PBKR03----[1] 19
443PEGUNIGALSIDASE ALFA[1] Pegunigalsidase alfa[1] D11685 --[1] 19
444Pegunigalsidase alpha----[1] 19
445Pending----[6] 13, 19, 96, 97, 113, 302
446Pentosan Polysulfat SP 54® injection solution----[1] 19
447Pentosan polysulfate[1] Pentosan polysulfate[1] D05428 --[3] 19, 26, 226
448PENTOSAN POLYSULFATE SODIUM[1] Pentosan polysulfate[1] D05428 --[1] 19
449Pentosan Polysulphate SP54----[1] 19
450Pentosan Polysulphate SP54®----[1] 19
451Perdix----[1] 19
452Plant cell expressed recombinant glucocerebrosidase (prGCD)[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
453Plant Cell Recombinant Glucocerebrosidase----[1] 19
454Plasma Lyso-GB3----[1] 19
455PLX-200----[1] 19
456PR001----[2] 6, 19
457Prednisone[1] Prednisone[1] D00473 [1] NR3C1 💬[1] Neuroactive ligand-receptor interaction 💬[48] 2, 6, 11, 13, 14, 19, 26, 35, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 53, 61, 63, 64, 65, 66, 79, 81, 84, 85, 86, 95, 96, 97, 113, 127, 162, 164, 205, 222, 224, 228, 251, 283, 284, 285, 288, 299, 300, 331
458Prevenar13----[2] 19, 265
459PrGCD[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
460PROCYSBI[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
461PRX-102----[1] 19
462PRX-102 (pegunigalsidase alfa)[1] Pegunigalsidase alfa[1] D11685 --[1] 19
463PRX-112----[1] 19
464PRX102----[1] 19
465PTC124----[4] 19, 113, 246, 299
466PYRIMETHAMINE[1] Pyrimethamine[1] D00488 --[4] 2, 4, 19, 65
467Quantitative chemical shift imaging (QCSI)----[1] 19
468RAAV 2/6 hIDS DONOR----[1] 19
469RAAV2/5-hNAGLU----[1] 19
470RAAV2/6 Left ZFN Vector----[1] 19
471RAAV2/6 Right ZFN Vector----[1] 19
472RAAV9.CMV.hNAGLU----[1] 19
473Radiation: Total Body Irradiation with Marrow Boosting----[2] 19, 234
474Rebisufligene etisparvovec----[1] 19
475Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A----[1] 19
476Recombinant alpha-galactosidase A[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
477RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)----[1] 19
478Recombinant human alpha-galactosidase----[1] 19
479Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL----[1] 19
480Recombinant human alpha-mannosidase----[1] 19
481Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)----[1] 19
482RECOMBINANT HUMAN ARYLSULFATASE A----[1] 19
483Recombinant Human Arylsulfatase A (rhASA)----[1] 19
484Recombinant Human Arylsulfatase A (rhASA,----[1] 19
485Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase----[1] 19
486RECOMBINANT HUMAN BETA GLUCURONIDASE----[1] 19
487Recombinant human beta-glucuronidase[1] Vestronidase alfa[1] D11004 [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
488Recombinant human beta-glucuronidase (rhGUS)[1] Vestronidase alfa[1] D11004 [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
489Recombinant human betaglucuronidase----[1] 19
490Recombinant human derived macrophage-targeted beta-Glucocerebrosidase----[1] 19
491Recombinant human derived macrophage-targeted ß-Glucocerebrosidase----[1] 19
492Recombinant human glucerebrosidase----[1] 19
493Recombinant human glucocerebrosidase----[1] 19
494Recombinant Human Glucocerebrosidase (prGCD)[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
495Recombinant human heparan N-sulfatase----[1] 19
496Recombinant human heparan N-sulfatase (rhHNS)----[1] 19
497Recombinant human heparan N-sulfatase [rhHNS]----[1] 19
498Recombinant human heparan Nsulfatase (rhHNS)----[1] 19
499Recombinant human lysosomal acid lipase----[1] 19
500Recombinant human lysosomal acid lipase (rhLAL)----[1] 19
501Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)[1] Cholesterol[1] D00040 --[1] 19
502Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] D00040 --[1] 19
503Recombinant human lysosomal alpha-mannosidase----[1] 19
504Recombinant human N-acetylgalactosamine 4-sulfatase----[1] 19
505Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110----[1] 19
506Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance----[1] 19
507Recombinant human N-acetylgalactosamine-6-sulfatase[1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
508Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)[1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
509Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110[1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
510Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance[1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
511Recombinant human Nacetylgalactosamine-----[1] 19
512Recombinant human tripeptidyl peptidase-1----[1] 19
513RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)----[1] 19
514Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene----[1] 19
515Reduced Intensity Conditioning----[2] 19, 65
516Reminder capsules----[1] 19
517Reminder Product----[1] 19
518REPLAGAL[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
519Replagal (Agalsidase Alfa)[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
520Replagal agalsidase alfa[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
521REPLAGAL ®[1] Agalsidase alfa[1] D02784 [1] GLA 💬[6] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome, Metabolic pathways, Sphingolipid metabolism 💬[1] 19
522Rh Growth Hormone[1] Somatotropin[1] D02691 [1] GHR 💬[5] Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬[1] 19
523RhARSA----[1] 19
524RhASA----[1] 19
525RhASA - Dose Level 1----[1] 19
526RhASA - Dose Level 2----[1] 19
527RhASA - Dose Level 3----[1] 19
528RhASM----[1] 19
529RhGALNS[1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
530RHGUS----[1] 19
531RhHNS-10 mg----[1] 19
532RhHNS-45 mg----[1] 19
533RhHNS-90 mg----[1] 19
534RhIDU (recombinant human-Alpha-L-Iduronidase)----[1] 19
535RhLAMAN[1] Velmanase alfa[1] D11024 [1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19
536RhNAGLU----[1] 19
537RhNAGLU radiolabelled----[1] 19
538RHNAGLU-IGF2----[1] 19
539Rimiducid[1] Rimiducid[1] D11195 [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
540Rituximab[1] Rituximab[1] D02994 [1] MS4A1 💬[1] Hematopoietic cell lineage 💬[49] 6, 11, 13, 14, 17, 19, 35, 36, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 53, 55, 56, 60, 61, 63, 64, 65, 66, 83, 84, 85, 86, 93, 94, 96, 97, 127, 162, 222, 229, 256, 271, 274, 283, 284, 285, 288, 296, 300, 331
541Rivogenlecleucel----[6] 19, 60, 65, 284, 285, 326
542RP 103----[2] 8, 19
543RP103----[2] 8, 19
544RVX000222----[1] 19
545SAF-301----[1] 19
546Saline Solution for Injection----[1] 19
547SAR402671, GZ402671 o GZ/SAR402671----[2] 19, 67
548SAR402671, GZ402671 or GZ / SAR402671----[1] 19
549SAR402671, GZ402671 or GZ/SAR402671----[3] 6, 19, 67
550SB-318----[1] 19
551SB-47171----[1] 19
552SB-47898----[1] 19
553SB-913----[1] 19
554SB-A6P-HRL Donor Vector----[1] 19
555SB-FIX----[1] 19
556SB-IDUA----[1] 19
557SBC-102[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
558SBC-102, recombinant human lysosomal acid lipase[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
559SBC-103----[1] 19
560ScAAV9.CB.CLN6----[1] 19
561ScAAV9.U1A.hSGSH----[1] 19
562ScAAV9.U1A.SGSH----[1] 19
563Sebelipasa Alfa[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
564Sebelipase Alfa[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
565Sebelipase alfa (SBC-102)[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
566Sebelipase alfa 0.35 mg/kg[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
567Sebelipase alfa 1 mg/kg[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
568Sebelipase alfa 3 mg/kg[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
569SHP611----[1] 19
570SHP675----[1] 19
571Sirolimus[1] Sirolimus[1] D00753 [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 💬[36] 6, 13, 15, 17, 19, 34, 35, 36, 46, 49, 51, 60, 61, 62, 63, 65, 66, 67, 84, 85, 89, 98, 127, 137, 157, 158, 192, 222, 277, 278, 279, 280, 281, 283, 285, 331
572Skeletal survey----[1] 19
573Skin biopsy----[2] 19, 84
574Slit Lamp assessment and intra-ocular pressure measurement----[1] 19
575SOBI003----[1] 19
576Sofosbuvir[1] Sofosbuvir[1] D10366 --[1] 19
577Somatropin[1] Somatotropin[1] D02691 [1] GHR 💬[5] Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬[19] 2, 3, 19, 46, 65, 74, 75, 78, 96, 107, 113, 187, 193, 195, 238, 265, 274, 276, 299
578Somatropin (DNA origin)[1] Somatotropin[1] D02691 [1] GHR 💬[5] Cytokine-cytokine receptor interaction, Growth hormone synthesis, secretion and action, JAK-STAT signaling pathway, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway 💬[1] 19
579Speech and modified barium swallow study[1] Barium---[1] 19
580Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride[1] Cysteamine[2] D03634 ,
D03635 		--[1] 19
581ST-920----[1] 19
582Stem Cell Transplant----[6] 19, 20, 60, 65, 164, 284
583Stem Cell Transplantation----[13] 13, 19, 20, 46, 51, 60, 62, 65, 90, 125, 164, 234, 326
584SUB195712----[1] 19
585Surgery to implant human CNS stem cells (HuCNS-SC)----[1] 19
586TALIGLUCERASE ALFA[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
587Taliglucerase alfa - Recombinant human glucocerebrosidase[1] Taliglucerase alfa[1] D09675 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
588TBD----[4] 19, 86, 276, 299
589Temperature sensitivity measurement with Advanced Thermal Stimulation----[1] 19
590Thiotepa[1] Thiotepa[1] D00583 --[12] 13, 19, 20, 28, 51, 60, 65, 85, 96, 164, 284, 285
591Thiotepa--escalated dose[1] Thiotepa[1] D00583 --[4] 19, 20, 65, 284
592Thiotepa--single daily dose[1] Thiotepa[1] D00583 --[4] 19, 20, 65, 284
593Thyroxine[1] Levothyroxine[2] D01010 ,
D08125 		[2] THRA,
THRB 💬		[2] Neuroactive ligand-receptor interaction, Thyroid hormone signaling pathway 💬[1] 19
594To be added after EV code is available----[1] 19
595Total body Irradiation----[6] 19, 20, 51, 60, 65, 285
596Trappsol Cyclo----[1] 19
597Trappsol® Cyclo™----[1] 19
598TREOSULFAN[1] Treosulfan[1] D07253 --[6] 13, 19, 62, 65, 283, 286
599Triapin----[1] 19
600Triapin& triapin Mite----[1] 19
601Tropicamide[1] Tropicamide[1] D00397 [5] CHRM1,
CHRM2,
CHRM3,
CHRM4,
CHRM5 💬		[13] Alzheimer disease, Calcium signaling pathway, Cholinergic synapse, Gastric acid secretion, Insulin secretion, Neuroactive ligand-receptor interaction, PI3K-Akt signaling pathway, Pancreatic secretion, Pathways of neurodegeneration - multiple diseases, Regulation of actin cytoskeleton, Salivary secretion, Taste transduction, cAMP signaling pathway 💬[2] 6, 19
602TSHA-101----[1] 19
603Unrelated Umbilical Cord Blood Transplant----[1] 19
604UPLYSO----[1] 19
605Urine collection----[1] 19
606USAN: sebelipasa alfa[1] Sebelipase alfa[1] D10377 [1] LIPA 💬[3] Cholesterol metabolism, Lysosome, Steroid biosynthesis 💬[1] 19
607USAN: sebelipase alpha----[1] 19
608UX003----[1] 19
609Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana----[1] 19
610VELAGLUCERASE ALFA[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
611Velaglucerase alfa (VPRIV)[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
612Velaglucerasi alfa----[1] 19
613VELMANASE ALFA[1] Velmanase alfa[1] D11024 [1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19
614Velmanase Alfa (e.g. Lamazym)[1] Velmanase alfa[1] D11024 [1] MAN2B1 💬[2] Lysosome, Other glycan degradation 💬[1] 19
615Venglustat[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67
616Venglustat (GZ402671)[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
617Venglustat GZ402671[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[2] 19, 67
618Venglustat malate[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 6, 19, 67
619Venglustat malato[1] Venglustat[1] D11633 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[2] 19, 67
620Vestronidase alfa[1] Vestronidase alfa[1] D11004 [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
621Vimizim® (elosulfase alfa)[1] Elosulfase alfa[1] D10333 [1] GALNS 💬[3] Glycosaminoglycan degradation, Lysosome, Metabolic pathways 💬[1] 19
622Visual field testing----[1] 19
623Vitamin D[1] Vitamin D---[24] 3, 13, 15, 19, 41, 43, 44, 46, 49, 50, 68, 93, 95, 96, 97, 98, 179, 222, 228, 235, 256, 274, 298, 299
624Vorinostat[1] Vorinostat[1] D06320 [11] HDAC1,
HDAC10,
HDAC11,
HDAC2,
HDAC3,
HDAC4,
HDAC5,
HDAC6,
HDAC7,
HDAC8,
HDAC9 💬		[17] Alcoholism, Amphetamine addiction, Amyotrophic lateral sclerosis, Apelin signaling pathway, Cell cycle, Chronic myeloid leukemia, Epstein-Barr virus infection, Human papillomavirus infection, Huntington disease, Longevity regulating pathway - multiple species, MicroRNAs in cancer, Neutrophil extracellular trap formation, Notch signaling pathway, Pathways in cancer, Thyroid hormone signaling pathway, Transcriptional misregulation in cancer, Viral carcinogenesis 💬[3] 19, 75, 96
625VPRIV[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
626VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
627VPRIV 400 Units powder for solution of infusion[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
628VPRIV ®,[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
629VPRIV®[1] Velaglucerase alfa[1] D09029 [1] GBA 💬[4] Lysosome, Metabolic pathways, Other glycan degradation, Sphingolipid metabolism 💬[1] 19
630VTS-270----[1] 19
631Warfarin[1] Warfarin[3] D00564 ,
D01280 ,
D08682 		[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 💬[13] 6, 8, 13, 19, 46, 49, 66, 85, 86, 88, 91, 96, 97
632Water[1] Water[1] D00001 --[23] 2, 5, 6, 13, 17, 19, 22, 35, 46, 51, 53, 56, 57, 63, 65, 67, 78, 84, 96, 97, 226, 278, 299
633Whole body Protein turnover----[4] 19, 46, 96, 299
634ZAVESCA[1] Miglustat[1] D05032 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[3] 19, 256, 299
635Zavesca (Miglustat)[1] Miglustat[1] D05032 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
636ZAVESCA 84CPS 100MG[1] Miglustat[1] D05032 [1] UGCG 💬[2] Metabolic pathways, Sphingolipid metabolism 💬[1] 19
637Zestoretic 10,20[1] Lisinopril[2] D00362 ,
D08131 		[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[1] 19
638ZESTORIC----[1] 19
639Zestril[1] Lisinopril[2] D00362 ,
D08131 		[1] ACE 💬[6] Chagas disease, Coronavirus disease - COVID-19, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[3] 19, 67, 222