Drug Description in Trials / DrugBank / KEGG DRUG

  

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No. Drug (Description in trials) DrugBank KEGG DRUG KEGG GENES KEGG PATHWAY Disease ID
11- Deoxygalactonojirimycin----[1] 19 💬
21-deoxygalactonojirimycin hydrochloride[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
312 lead electrocardiogram----[1] 19 💬
412S-IT----[1] 19 💬
515-0 H20----[2] 6, 19 💬
61629063-78-0----[1] 19 💬
72(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID----[1] 19 💬
82-hour Holter Monitor----[1] 19 💬
94D-310[1] 4D-310---[1] 19 💬
10500 0500 0----[1] 19 💬
1189 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)----[1] 19 💬
1289Zr-SBC-103----[1] 19 💬
13[045251016]----[1] 19 💬
14[14C] AT1001----[1] 19 💬
15[14C]-Genz-112638----[1] 19 💬
16[A16AB04]----[1] 19 💬
17[AAV2/8.TBG.hARSB]----[1] 19 💬
18[ACT-434964]----[1] 19 💬
19[AT1001]----[1] 19 💬
20[difenidramina cloridrato]----[1] 19 💬
21[DNL310]----[1] 19 💬
22[FLT190][1] FLT190---[1] 19 💬
23[GZ402671]----[1] 19 💬
24[HGT-1410]----[1] 19 💬
25[JR-141]----[1] 19 💬
26[NA]----[34] 3, 6, 11, 13, 14, 19, 28, 35, 41, 46, 49, 50, 53, 57, 62, 63, 65, 75, 79, 85, 90, 96, 97, 98, 113, 127, 162, 195, 223, 229, 251, 294, 299, 302 💬
27[OTL-200]----[1] 19 💬
28[Paracetamolo]----[4] 14, 19, 300, 309 💬
29[Prednisone][1] Prednisone[1] Prednisone 💬 [1] NR3C1 💬 [1] Neuroactive ligand-receptor interaction 💬[5] 19, 66, 90, 300, 303 💬
30[PRX-102]----[1] 19 💬
31[rhLAMAN][1] Velmanase alfa[1] Velmanase alfa 💬 [1] MAN2B1 💬 [2] Lysosome biogenesis, Other glycan degradation 💬[1] 19 💬
32[SAR402671, GZ402671 o GZ/SAR402671]----[2] 19, 67 💬
33[SAR402671, GZ402671 or GZ / SAR402671]----[1] 19 💬
34[SHP611]----[1] 19 💬
35[ST-920]----[1] 19 💬
36A (migalastat)[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
37A16AX06----[1] 19 💬
38AAV2/8.TBG.hARSB----[1] 19 💬
39AAV2CUhCLN2 (3x10^12 particle units)----[1] 19 💬
40AAV9-CAG-coh-SGSH----[1] 19 💬
41AAV9-GLB1----[1] 19 💬
42AAVrh.10-hMPSIIIA----[1] 19 💬
43AAVrh.10cuARSA----[1] 19 💬
44AAVrh.10CUCLN2----[1] 19 💬
45AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies----[1] 19 💬
46AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies----[1] 19 💬
47Abatacept[1] Abatacept[1] Abatacept 💬 [2] CD80,
CD86 💬 		[14] Allograft rejection, Autoimmune thyroid disease, Cell adhesion molecule (CAM) interaction, Graft-versus-host disease, IgSF CAM signaling, 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 💬[28] 2, 11, 13, 19, 41, 44, 46, 49, 50, 51, 53, 55, 60, 65, 84, 93, 95, 96, 97, 107, 160, 164, 222, 223, 271, 284, 285, 300 💬
48Abcertin----[1] 19 💬
49Abdominal ultrasound----[2] 19, 231 💬
50ABO-101----[2] 19, 234 💬
51ABO-102[1] Rebisufligene etisparvovec---[1] 19 💬
52Accupro----[1] 19 💬
53Accuretic----[1] 19 💬
54Acetaminophen[1] Acetaminophen[1] Acetaminophen 💬 [2] PTGS1,
PTGS2 💬 		[23] Alzheimer disease, Arachidonic acid metabolism, C-type lectin receptor signaling pathway, Chemical carcinogenesis - DNA adducts, Efferocytosis, Human cytomegalovirus infection, Human papillomavirus infection, IL-17 signaling pathway, Kaposi sarcoma-associated herpesvirus infection, Leishmaniasis, MicroRNAs in cancer, NF-kappa B signaling pathway, Ovarian steroidogenesis, Oxytocin signaling pathway, Pathways in cancer, Pathways of neurodegeneration - multiple diseases, Platelet activation, Regulation of lipolysis in adipocytes, Retrograde endocannabinoid signaling, Serotonergic synapse, Small cell lung cancer, TNF signaling pathway, VEGF signaling pathway 💬[19] 11, 13, 19, 34, 35, 46, 49, 51, 63, 70, 77, 86, 96, 222, 231, 256, 271, 300, 337 💬
55Acetylcysteine[1] Acetylcysteine[1] Acetylcysteine 💬 --[25] 2, 6, 13, 19, 20, 21, 34, 49, 51, 53, 58, 63, 85, 90, 94, 97, 111, 164, 193, 233, 296, 298, 299, 303, 337 💬
56Acetylcysteine amide[2] Acetylcysteine,
Acetylcysteine amide		[1] Acetylcysteine 💬 --[3] 19, 90, 303 💬
57ACT-434964----[1] 19 💬
58Active comparator (Agalsidase beta)[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
59Adalimumab[1] Adalimumab[1] Adalimumab 💬 [1] TNF 💬 [66] AGE-RAGE signaling pathway in diabetic complications, Adipocytokine signaling pathway, African trypanosomiasis, Alcoholic liver disease, Allograft rejection, Alzheimer disease, Amoebiasis, Amyotrophic lateral sclerosis, Antigen processing and presentation, Apoptosis, Asthma, C-type lectin receptor signaling pathway, Chagas disease, Coronavirus disease, 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 💬[22] 19, 37, 38, 40, 41, 46, 55, 56, 84, 90, 96, 97, 107, 151, 160, 162, 164, 222, 226, 269, 270, 271 💬
60Adalimumab Injection [Humira][1] Adalimumab[1] Adalimumab 💬 [1] TNF 💬 [66] AGE-RAGE signaling pathway in diabetic complications, Adipocytokine signaling pathway, African trypanosomiasis, Alcoholic liver disease, Allograft rejection, Alzheimer disease, Amoebiasis, Amyotrophic lateral sclerosis, Antigen processing and presentation, Apoptosis, Asthma, C-type lectin receptor signaling pathway, Chagas disease, Coronavirus disease, 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 💬
61ADENO-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] 19 💬
62Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene----[1] 19 💬
63Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-betagal)----[1] 19 💬
64Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal)----[1] 19 💬
65Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA----[1] 19 💬
66Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector----[1] 19 💬
67Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector----[1] 19 💬
68Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector----[1] 19 💬
69Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector----[1] 19 💬
70Adeno-associated virus serotype hu68 containing the human GALC gene----[1] 19 💬
71Adeno-associated virus serotype rh.10 expressing beta-galactosidase----[1] 19 💬
72Adjuvant IM Therapy----[1] 19 💬
73Adjuvant Immunomodulatory (IM) Therapy----[1] 19 💬
74Administration Kit----[1] 19 💬
75Adrabetadex----[1] 19 💬
76Afegostat[1] Afegostat[2] Afegostat ,
Afegostat tartrate 💬 		--[1] 19 💬
77Afegostat tartrate[1] Afegostat[2] Afegostat ,
Afegostat tartrate 💬 		--[1] 19 💬
78Agalsidase----[1] 19 💬
79AGALSIDASE ALFA[1] Agalsidase alfa[1] Agalsidase alfa 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
80Agalsidase Alpha----[1] 19 💬
81AGALSIDASE BETA[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
82AGALSIDASE BETA (GZ419828)[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
83Agalsidase beta (recombinant form)[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
84Agalsidase beta from Biosidus 1 mg/kg[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
85AGALSIDASI ALFA----[1] 19 💬
86AGALSIDASI BETA----[1] 19 💬
87AGT-181----[1] 19 💬
88AGT-181 (HIRMAb-IDUA)----[1] 19 💬
89AGT-182[1] AGT-182---[1] 19 💬
90AL01211----[2] 19, 67 💬
91Aldurazyme----[1] 19 💬
92Aldurazyme (laronidase)[1] Laronidase[1] Laronidase 💬 [1] IDUA 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[2] 19, 256 💬
93Aldurazyme (Recombinant Human Alpha-L-Iduronidase)----[1] 19 💬
94ALDURAZYME*INFUS 1FL 5ML 500U----[1] 19 💬
95Aldurazyme®----[1] 19 💬
96ALEMTUZUMAB[1] Alemtuzumab[1] Alemtuzumab 💬 --[21] 13, 14, 15, 19, 20, 36, 43, 44, 46, 51, 60, 63, 65, 85, 96, 107, 164, 283, 284, 285, 326 💬
97Alendronate sodium[1] Alendronic acid[2] Alendronate sodium ,
Alendronic acid 💬 		[1] FDPS 💬 [3] Human T-cell leukemia virus 1 infection, Influenza A, Terpenoid backbone biosynthesis 💬[4] 19, 46, 223, 299 💬
98Alfa-manosidadas humana recombinante----[1] 19 💬
99ALISERIN - 25 MG GRANULATO EFFERVESCENTE 20 BUSTINE----[1] 19 💬
100Allogeneic stem cell transplantation----[5] 19, 20, 49, 62, 84 💬
101Ambroxol[1] Ambroxol[2] Ambroxol hydrochloride ,
Ambroxol 💬 		--[3] 2, 6, 19 💬
102Ambroxol Hydrochloride[1] Ambroxol[2] Ambroxol hydrochloride ,
Ambroxol 💬 		--[2] 6, 19 💬
103Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day[1] Ambroxol[2] Ambroxol hydrochloride ,
Ambroxol 💬 		--[1] 19 💬
104Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day[1] Ambroxol[2] Ambroxol hydrochloride ,
Ambroxol 💬 		--[1] 19 💬
105Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day[1] Ambroxol[2] Ambroxol hydrochloride ,
Ambroxol 💬 		--[1] 19 💬
106AMBROXOL HYDROCHLORIDE PH. EUR.[1] Ambroxol[2] Ambroxol hydrochloride ,
Ambroxol 💬 		--[1] 19 💬
107AMT-191----[1] 19 💬
108Anakinra[1] Anakinra[1] Anakinra 💬 [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 💬[22] 2, 13, 15, 19, 28, 37, 41, 46, 49, 50, 54, 56, 57, 84, 86, 97, 106, 107, 149, 153, 266, 299 💬
109Antihistamine----[3] 13, 19, 46 💬
110Antipyretic----[2] 19, 257 💬
111ARIMOCLOMOL[1] Arimoclomol[2] Arimoclomol ,
Arimoclomol citrate 💬 		--[3] 2, 15, 19 💬
112Arimoclomol (hard gelatine capsule)[1] Arimoclomol[2] Arimoclomol ,
Arimoclomol citrate 💬 		--[1] 19 💬
113Arimoclomol (HPMC capsule)[1] Arimoclomol[2] Arimoclomol ,
Arimoclomol citrate 💬 		--[1] 19 💬
114Arimoclomol citrate[1] Arimoclomol[2] Arimoclomol ,
Arimoclomol citrate 💬 		--[2] 15, 19 💬
115Arylsulfatase A----[1] 19 💬
116AT-GTX-501----[1] 19 💬
117AT1001----[1] 19 💬
118AT1001 150 mg----[1] 19 💬
119AT2101----[1] 19 💬
120ATALUREN[1] Ataluren[1] Ataluren 💬 --[6] 19, 113, 140, 246, 299, 329 💬
121ATG----[6] 13, 19, 60, 96, 222, 285 💬
122AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE----[1] 19 💬
123Autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA----[1] 19 💬
124Autologous Plasmablasts----[1] 19 💬
125Autologous Plasmablasts (B cells)----[1] 19 💬
126AVR-RD-01[1] AVR-RD-01---[1] 19 💬
127AVR-RD-02[1] AVR-RD-02---[1] 19 💬
128AX 250----[1] 19 💬
129AXO-AAV-GM2----[1] 19 💬
130AXO-AAV-GM2 High Dose----[1] 19 💬
131AXO-AAV-GM2 Low Dose----[1] 19 💬
132AXO-AAV-GM2 Middle Dose----[1] 19 💬
133AXO-AAV-GM2 Starting Dose----[1] 19 💬
134AZ-3102----[1] 19 💬
135AZ-3102 (Dose 1)----[1] 19 💬
136AZ-3102 (Dose 2)----[1] 19 💬
137Azathioprine[1] Azathioprine[2] Azathioprine ,
Azathioprine sodium 💬 		--[26] 6, 11, 13, 19, 35, 42, 43, 44, 45, 46, 49, 55, 57, 63, 65, 66, 84, 85, 93, 94, 95, 96, 97, 162, 223, 300 💬
138Azathioprine (AZA)[1] Azathioprine[2] Azathioprine ,
Azathioprine sodium 💬 		--[5] 13, 19, 49, 55, 97 💬
139B (migalastat)[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
140Barium[1] Barium---[5] 2, 6, 19, 97, 98 💬
141Behavioral: Neurocognitive testing----[1] 19 💬
142Behavioral: PICC or other Central line placement----[1] 19 💬
143Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
144Biological assays----[1] 19 💬
145Blood draw----[16] 3, 6, 13, 19, 46, 48, 49, 51, 58, 65, 67, 193, 215, 231, 298, 299 💬
146Blood sample----[33] 2, 3, 6, 8, 10, 13, 14, 19, 34, 46, 48, 49, 51, 53, 61, 63, 65, 66, 67, 77, 79, 84, 88, 96, 97, 107, 113, 127, 195, 206, 235, 274, 299 💬
147Blood sampling for dosage----[1] 19 💬
148BM110----[1] 19 💬
149BMN 110----[1] 19 💬
150BMN 110 - Every Other Week----[1] 19 💬
151BMN 110 - Weekly----[1] 19 💬
152BMN 110 Every Other Week----[1] 19 💬
153BMN 110 Weekly----[1] 19 💬
154BMN 190----[1] 19 💬
155BMN 250----[1] 19 💬
156BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)----[1] 19 💬
157Brain MRI/MRS/fMRI----[1] 19 💬
158Brineura----[1] 19 💬
159BUSILVEX----[4] 19, 36, 60, 65 💬
160BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI----[2] 19, 65 💬
161Busulfan[1] Busulfan[1] Busulfan 💬 --[14] 19, 20, 28, 34, 36, 49, 51, 60, 65, 164, 274, 284, 285, 326 💬
162Busulfan, Cyclophosphamide, Antithymocyte Globulin[2] Busulfan,
Cyclophosphamide		[3] Busulfan ,
Cyclophosphamide ,
Cyclophosphamide 💬 		--[2] 19, 20 💬
163Busulfan, Cyclophosphamide, ATG[2] Busulfan,
Cyclophosphamide		[3] Busulfan ,
Cyclophosphamide ,
Cyclophosphamide 💬 		--[1] 19 💬
164BUSULFANO----[1] 19 💬
165C (migalastat)[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
166Calcium[1] Calcium---[39] 2, 3, 6, 13, 15, 19, 46, 48, 49, 51, 53, 56, 62, 67, 68, 71, 74, 75, 79, 84, 90, 93, 94, 95, 96, 97, 156, 186, 214, 215, 222, 224, 235, 256, 265, 271, 274, 298, 299 💬
167Calcium carbonate[2] Calcium,
Calcium carbonate		[2] Calcium carbonate ,
Calcium carbonate 💬 		--[10] 13, 19, 46, 53, 71, 75, 96, 97, 235, 299 💬
168CALD HR-D (High-Risk, Regimen C)----[5] 19, 20, 125, 234, 326 💬
169CALD HR-D (High-Risk, Regimen D)----[5] 19, 20, 125, 234, 326 💬
170CALD SR-A (Standard-Risk, Regimen A)----[5] 19, 20, 125, 234, 326 💬
171CALD SR-B (Standard-Risk, Regimen B)----[5] 19, 20, 125, 234, 326 💬
172Campath-1H----[6] 19, 20, 60, 65, 234, 326 💬
173Cannabidiol[1] Cannabidiol[1] Cannabidiol 💬 [2] CNR1,
CNR2 💬 		[4] Neuroactive ligand-receptor interaction, Rap1 signaling pathway, Retrograde endocannabinoid signaling, Thermogenesis 💬[20] 6, 8, 13, 19, 36, 46, 51, 95, 96, 97, 140, 144, 145, 156, 157, 158, 193, 206, 226, 271 💬
174Cannabidiol oral solution (Epidyolex)[1] Cannabidiol[1] Cannabidiol 💬 [2] CNR1,
CNR2 💬 		[4] Neuroactive ligand-receptor interaction, Rap1 signaling pathway, Retrograde endocannabinoid signaling, Thermogenesis 💬[3] 19, 158, 206 💬
175Cannabis sativa L., folium cum flore----[1] 19 💬
176Capsaicin[1] Capsaicin[1] Capsaicin 💬 [1] TRPV1 💬 [2] Inflammatory mediator regulation of TRP channels, Neuroactive ligand-receptor interaction 💬[5] 2, 6, 19, 86, 226 💬
177Cebsulfase alfa----[1] 19 💬
178Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi----[1] 19 💬
179Cerdelga----[1] 19 💬
180CEREDASE™----[1] 19 💬
181CEREZYME----[1] 19 💬
182Cerezyme (imiglucerase for injection)[1] Imiglucerase[2] Alglucerase ,
Imiglucerase 💬 		[1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
183Cerezyme 400 Units Powder for concentrate for solution for infusion----[1] 19 💬
184Cerezyme 400 Units Powder for concentrate for solution for infusionPharmaceutical Form: Powder for concentrate for solution for infusionINN or Proposed INN: ImigluceraseCAS Number: 154248-97-2Concentration unit: U unit(s)Concentration type: equalConcentration number: 400-Pharmaceutical form of the placebo: Solution for infusionRoute of administration of the placebo: Intravenous use----[1] 19 💬
185Cerezyme 400 Unità Polvere per concentrato per soluzione per infusione----[1] 19 💬
186Cerezyme®----[1] 19 💬
187Cerezyme® / Imiglucerase[1] Imiglucerase[2] Alglucerase ,
Imiglucerase 💬 		[1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
188Cerliponase Alfa[1] Cerliponase alfa[1] Cerliponase alfa 💬 [1] TPP1 💬 [1] Lysosome biogenesis 💬[1] 19 💬
189Cetirizine[1] Cetirizine[2] Cetirizine hydrochloride ,
Cetirizine 💬 		[1] HRH1 💬 [4] Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction 💬[3] 13, 19, 35 💬
190CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE----[1] 19 💬
191CHF6657----[1] 19 💬
192CHOLECALCIFEROL[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 💬[10] 13, 19, 34, 46, 49, 96, 97, 222, 298, 299 💬
193Cholesterol[1] Cholesterol[1] Cholesterol 💬 --[10] 8, 19, 71, 79, 90, 160, 222, 259, 261, 310 💬
194CliniMACS® TCR a/beta Reagent Kit and CliniMACS® CD19 System----[3] 19, 96, 222 💬
195CliniMACS® TCR a/ß Reagent Kit and CliniMACS® CD19 System----[3] 19, 96, 222 💬
196Clofarabine[1] Clofarabine[1] Clofarabine 💬 [3] POLA1,
POLA2,
RRM1 💬 		[5] DNA replication, Drug metabolism - other enzymes, Glutathione metabolism, Purine metabolism, Pyrimidine metabolism 💬[9] 19, 20, 40, 46, 49, 60, 96, 234, 326 💬
197Contrast sensitivity measurement----[1] 19 💬
198Coversyl----[2] 19, 167 💬
199Coversyl PLUS----[1] 19 💬
200CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE----[1] 19 💬
201Cyclophosphamide[1] Cyclophosphamide[2] Cyclophosphamide ,
Cyclophosphamide 💬 		--[50] 11, 13, 14, 16, 19, 20, 26, 28, 34, 35, 36, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 52, 53, 56, 60, 61, 62, 63, 65, 66, 84, 93, 96, 162, 164, 172, 205, 221, 222, 223, 224, 256, 265, 274, 283, 284, 285, 288, 326, 331 💬
202Cyclophosphamide 100 mg/Kg[1] Cyclophosphamide[2] Cyclophosphamide ,
Cyclophosphamide 💬 		--[2] 19, 222 💬
203Cyclophosphamide 1200 mg/Kg[1] Cyclophosphamide[2] Cyclophosphamide ,
Cyclophosphamide 💬 		--[2] 19, 222 💬
204Cyclosporine[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 💬[39] 11, 14, 19, 20, 28, 36, 40, 41, 42, 45, 46, 49, 50, 53, 56, 58, 60, 61, 62, 63, 64, 65, 67, 90, 95, 97, 107, 118, 162, 164, 222, 223, 226, 228, 234, 283, 284, 285, 302 💬
205Cyclosporine 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 💬[12] 19, 20, 36, 49, 50, 53, 58, 60, 107, 228, 234, 283 💬
206Cyclosporine 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 💬
207Cystadane anhydrous----[1] 19 💬
208CYSTADROPS----[1] 19 💬
209CYSTADROPS 0.55% eye drops, solution----[1] 19 💬
210CYSTAGON----[3] 8, 19, 299 💬
211Cystagon 150mg----[1] 19 💬
212Cystagon 50mg----[1] 19 💬
213Cystagon®----[1] 19 💬
214Cystagon® (Cysteamine Bitartrate)[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
215Cysteamine[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[4] 8, 19, 21, 299 💬
216Cysteamine (mercaptamine) viscous solution[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
217Cysteamine Bitartrate[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[4] 8, 19, 21, 299 💬
218Cysteamine bitartrate (INN: mercaptamine bitartrate[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
219Cysteamine bitartrate (INN: mercaptamine bitartrate )[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
220Cysteamine bitartrate (INN: mercaptamine bitartrate)[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
221Cysteamine Bitartrate Delayed-release Capsules[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
222Cysteamine Bitartrate Delayed-release Capsules (RP103)[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
223CYSTEAMINE HYDROCHLORIDE[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
224Cysteine[1] Cysteine[2] L-Cysteine ,
Cysteine hydrochloride 💬 		--[13] 6, 13, 19, 34, 60, 84, 85, 88, 90, 254, 296, 310, 337 💬
225D (migalastat)[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
226Dapagliflozin[1] Dapagliflozin[1] Dapagliflozin 💬 --[16] 6, 19, 28, 40, 41, 49, 57, 66, 67, 86, 88, 97, 210, 215, 218, 222 💬
227Dapagliflozin 10mg Tab[1] Dapagliflozin[1] Dapagliflozin 💬 --[4] 19, 97, 218, 222 💬
228DEFLEGMIN 75mg, kapsulki o przedluzonym uwalnianiu----[1] 19 💬
229DEXA scan----[3] 19, 46, 96 💬
230Dexamethasone[1] Dexamethasone[3] Dexamethasone ,
Dexamethasone sodium phosphate ,
Dexamethasone phosphate 💬 		[1] NR3C1 💬 [1] Neuroactive ligand-receptor interaction 💬[29] 13, 14, 16, 19, 28, 35, 46, 53, 63, 64, 65, 70, 71, 74, 75, 81, 84, 90, 96, 97, 162, 222, 223, 235, 257, 283, 296, 299, 331 💬
231Diagnostic Test: 1 hour ECG----[1] 19 💬
232Diagnostic Test: 24 hour pulse wave analysis----[1] 19 💬
233Diagnostic Test: 24-hour blood pressure measurement----[1] 19 💬
234Diagnostic Test: 68Ga-DOTATATE PET/MRI[1] Dotatate---[1] 19 💬
235Diagnostic Test: Ambulatory blood pressure monitoring----[1] 19 💬
236Diagnostic Test: Audiology assessment with ABR----[1] 19 💬
237Diagnostic Test: Biochemistry and immune phenotyping----[1] 19 💬
238Diagnostic Test: Blood sample collection----[2] 19, 20 💬
239Diagnostic Test: blood sampling (alpha-Galactosidase & LysoGb3)----[2] 19, 58 💬
240Diagnostic Test: Bone density scan (DEXA)----[1] 19 💬
241Diagnostic Test: Cardiac MRI, ECG/Holter and Blood Biomarkers----[1] 19 💬
242Diagnostic Test: Cardio MRI----[1] 19 💬
243Diagnostic Test: Cardiopulmonary exercise testing (CPET)----[1] 19 💬
244Diagnostic Test: dry blood test----[2] 19, 97 💬
245Diagnostic Test: Dynamic retinal vessel analysis (DVA)----[1] 19 💬
246Diagnostic Test: Echocardiogram----[2] 19, 86 💬
247Diagnostic Test: Echocardiography----[2] 19, 58 💬
248Diagnostic Test: Echocardiography at M24----[1] 19 💬
249Diagnostic Test: Echocardiography at T0----[1] 19 💬
250Diagnostic Test: Electrocardiogram (EKG)----[1] 19 💬
251Diagnostic Test: Exercise test----[1] 19 💬
252Diagnostic Test: Laboratory tests----[1] 19 💬
253Diagnostic Test: Measures of arterial stiffness and endothelial function----[1] 19 💬
254Diagnostic Test: Ophthalmological consultation----[1] 19 💬
255Diagnostic Test: Optical coherence tomography (OCT)----[1] 19 💬
256Diagnostic Test: Questionnaires (Patient reported outcomes)----[1] 19 💬
257Diagnostic Test: Serum and urine biomarkers----[1] 19 💬
258Diagnostic Test: Static retinal vessel analysis (SVA)----[1] 19 💬
259DIFENIDRAMINA CLORIDRATO----[3] 11, 19, 63 💬
260Diphenhydramine[1] Diphenhydramine[4] Diphenhydramine ,
Diphenhydramine hydrochloride ,
Diphenhydramine salicylate ,
Diphenhydramine citrate 💬 		[1] HRH1 💬 [4] Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction 💬[12] 11, 13, 19, 49, 51, 63, 70, 86, 96, 222, 256, 300 💬
261DNL126----[1] 19 💬
262DNL310----[1] 19 💬
263DNL310 Drug Substance----[1] 19 💬
264Dotatate[1] Dotatate---[5] 19, 41, 75, 84, 238 💬
265Dried blood spot (DBS) sampling----[1] 19 💬
266DRX005B----[1] 19 💬
267DUOC-01----[3] 13, 19, 20 💬
268E (migalastat)[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
269Echocardiogram----[3] 19, 215, 216 💬
270ELAPRASE----[1] 19 💬
271Elaprase 2mg/ml----[1] 19 💬
272Elaprase for intravenous (IV) infusion----[1] 19 💬
273Elaprase_0.5mg/kg----[1] 19 💬
274Elelyso----[1] 19 💬
275Elelyso 60 units/kg----[1] 19 💬
276Elfabrio----[1] 19 💬
277Eliglustat[1] Eliglustat[2] Eliglustat ,
Eliglustat tartrate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
278Eliglustat GZ385660[1] Eliglustat[2] Eliglustat ,
Eliglustat tartrate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
279ELIGLUSTAT TARTRATE[1] Eliglustat[2] Eliglustat ,
Eliglustat tartrate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
280Eliglustat Tartrate Capsules[1] Eliglustat[2] Eliglustat ,
Eliglustat tartrate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
281Eliglustat Tartrate – Powder for Oral Suspension[1] Eliglustat[2] Eliglustat ,
Eliglustat tartrate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
282Elosulfase alfa[1] Elosulfase alfa[1] Elosulfase alfa 💬 [1] GALNS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
283Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM][1] Elosulfase alfa[1] Elosulfase alfa 💬 [1] GALNS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
284ELX-02[1] ELX-02---[3] 19, 218, 299 💬
285Enalapril[1] Enalapril[2] Enalapril maleate ,
Enalapril 💬 		[1] ACE 💬 [6] Chagas disease, Coronavirus disease, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[10] 13, 19, 46, 57, 66, 113, 210, 222, 223, 279 💬
286Enalapril and other angiotensin converting enzyme inhibitors[1] Enalapril[2] Enalapril maleate ,
Enalapril 💬 		[1] ACE 💬 [6] Chagas disease, Coronavirus disease, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[1] 19 💬
287Enriched Hematopoetic Stem Cell Infusion----[2] 19, 284 💬
288Enzyme replacement----[1] 19 💬
289Enzyme Replacement Agent----[2] 19, 256 💬
290Enzyme replacement therapy----[1] 19 💬
291Enzyme replacement therapy (Agalsidase alpha (Replagal®))----[1] 19 💬
292Enzyme replacement therapy (ERT)----[1] 19 💬
293Epidiolex----[1] 19 💬
294ERT----[1] 19 💬
295EU-sourced Cerezyme----[1] 19 💬
296EXG110 Injection----[1] 19 💬
297Fabagal® (Agalsidase beta)[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
298FABRAZYME----[1] 19 💬
299Fabrazyme (agalsidase beta)[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
300Fabrazyme (agalsidase beta) 1 mg/kg[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
301FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO----[1] 19 💬
302Fabrazyme 35 mg----[1] 19 💬
303Fabrazyme 5 mg----[1] 19 💬
304FBX-101[1] FBX-101---[1] 19 💬
305Fexofenadine[1] Fexofenadine[2] Fexofenadine hydrochloride ,
Fexofenadine 💬 		[1] HRH1 💬 [4] Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction 💬[4] 6, 13, 19, 46 💬
306FLT190[1] FLT190---[1] 19 💬
307FLT201[1] FLT201---[1] 19 💬
308FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML----[1] 19 💬
309FLUDARABINA FOSFATO----[1] 19 💬
310Fludarabine[1] Fludarabine[2] Fludarabine phosphate ,
Fludarabine 💬 		[1] RRM1 💬 [4] Drug metabolism - other enzymes, Glutathione metabolism, Purine metabolism, Pyrimidine metabolism 💬[30] 11, 13, 14, 16, 19, 28, 34, 35, 36, 44, 46, 49, 50, 51, 60, 61, 62, 63, 65, 85, 96, 107, 164, 222, 223, 283, 284, 285, 286, 326 💬
311Fluorescein[1] Fluorescein[2] Fluorescein ,
Fluorescein sodium 💬 		--[4] 19, 22, 41, 97 💬
312GA-GCB[1] Velaglucerase alfa[1] Velaglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
313GA-GCB (velaglucerase alfa)[1] Velaglucerase alfa[1] Velaglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
314GABAPENTIN[1] Gabapentin[1] Gabapentin 💬 [5] CACNA2D1,
CACNA2D2,
CACNA2D3,
CACNA2D4,
SLC6A1 💬 		[10] Adrenergic signaling in cardiomyocytes, Arrhythmogenic right ventricular cardiomyopathy, Cardiac muscle contraction, Dilated cardiomyopathy, GABAergic synapse, Hypertrophic cardiomyopathy, MAPK signaling pathway, Neuroactive ligand signaling, Oxytocin signaling pathway, Synaptic vesicle cycle 💬[7] 6, 13, 18, 19, 70, 226, 298 💬
315Galafold----[1] 19 💬
316GALSULFASE[1] Galsulfase[1] Galsulfase 💬 [1] ARSB 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
317GC1111_0.5mg/kg----[1] 19 💬
318GC1111_1.0mg/kg----[1] 19 💬
319GC1119----[1] 19 💬
320GC1123----[1] 19 💬
321GC1130A----[1] 19 💬
322Gene Activated a-Galactosidase A----[1] 19 💬
323Gene Activated Human Glucocerebrosidase----[1] 19 💬
324Gene activated human glucocerebrosidase, velaglucerase alfa[1] Velaglucerase alfa[1] Velaglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
325Gene therapy----[2] 6, 19 💬
326Gene Transfer with an AAV9 Vector Expressing Human beta-galactosidase----[1] 19 💬
327Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase----[1] 19 💬
328Gene-Activated Human Glucocerebrosidase----[1] 19 💬
329Gene-Activated Human Glucocerebrosidase 200U/vial----[1] 19 💬
330Gene-Activated Human Glucocerebrosidase 400U/vial----[1] 19 💬
331Genetic HMI-203[1] HMI-203---[1] 19 💬
332Genetic: 26 common Fabry mutation types in Taiwan----[1] 19 💬
333Genetic: Active Comparator: Allo-HSCT----[1] 19 💬
334Genetic: AT-GTX-501----[1] 19 💬
335Genetic: Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII----[1] 19 💬
336Genetic: BBM-F101 injection----[1] 19 💬
337Genetic: Blood sampling for biological and genetic analysis----[1] 19 💬
338Genetic: CTNS-RD-04 or CTNS-RD-04-LB (where the suffix -LB stands for LentiBOOST)----[1] 19 💬
339Genetic: DFT383----[1] 19 💬
340Genetic: Diagnosis of Fabry disease----[1] 19 💬
341Genetic: EXG110 injection----[1] 19 💬
342Genetic: Experimental: OTL-203[1] OTL-203---[1] 19 💬
343Genetic: FLT190[1] FLT190---[1] 19 💬
344Genetic: FLT201[1] FLT201---[1] 19 💬
345Genetic: Frozen 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 💬
346Genetic: genetic analyses----[1] 19 💬
347Genetic: GLA gene----[1] 19 💬
348Genetic: High dose CLN-301----[1] 19 💬
349Genetic: human glucocerebrosidase cDNA----[1] 19 💬
350Genetic: human glucocerebrosidase gene into autologous peripheral blood stem cells----[1] 19 💬
351Genetic: intracerebral administration of AAVrh.10cuARSA----[1] 19 💬
352Genetic: Intrathecal and intravenous LV gene therapy----[2] 19, 20 💬
353Genetic: JWK008 Single intravenous infusion administration----[1] 19 💬
354Genetic: Lentiviral TYF-ARSA correction of patient's autologous HSCs----[1] 19 💬
355Genetic: Low dose CLN-301----[1] 19 💬
356Genetic: LY-M001----[1] 19 💬
357Genetic: LY3884961[1] LY3884961---[1] 19 💬
358Genetic: lymphocyte gene therapy----[1] 19 💬
359Genetic: LYS-GM101----[1] 19 💬
360Genetic: NGN-101[1] NGN-101---[1] 19 💬
361Genetic: No Intervention----[2] 19, 113 💬
362Genetic: OTL-200----[1] 19 💬
363Genetic: OTL-200 Gene Therapy----[1] 19 💬
364Genetic: RGX-111[1] RGX-111---[1] 19 💬
365Genetic: RGX-121[1] RGX-121---[1] 19 💬
366Genetic: RGX-121-3102[1] RGX-121---[1] 19 💬
367Genetic: SAF-301----[1] 19 💬
368Genetic: scAAV9/AGA----[1] 19 💬
369Genetic: transduced CD34+ hematopoietic stem cell----[2] 19, 20 💬
370Genetic: TTX-381----[1] 19 💬
371GENISTEIN[1] Genistein[1] Genistein 💬 --[2] 19, 299 💬
372Genistein aglycone[1] Genistein[1] Genistein 💬 --[1] 19 💬
373Genz-112638----[1] 19 💬
374Genz-682452-AA----[1] 19 💬
375Genz-682452-AU----[3] 6, 19, 67 💬
376GLUCOCEREBROSIDASE----[1] 19 💬
377GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO----[1] 19 💬
378GNR-055----[1] 19 💬
379GNR-055 1.0-2.0-3.0 mg/kg----[1] 19 💬
380GNR-055 2.0 mg/kg----[1] 19 💬
381GNR-055 3.0 mg/kg----[1] 19 💬
382Gold[1] Gold---[71] 2, 5, 6, 11, 13, 17, 19, 21, 22, 28, 34, 35, 46, 49, 50, 53, 54, 56, 57, 58, 60, 63, 64, 65, 66, 68, 71, 75, 76, 77, 79, 84, 85, 86, 88, 91, 93, 95, 96, 97, 98, 107, 113, 127, 137, 158, 162, 167, 171, 193, 206, 215, 218, 222, 223, 224, 226, 227, 228, 230, 235, 271, 291, 294, 296, 298, 299, 310, 318, 331, 340 💬
383GR181413----[1] 19 💬
384GR181413A----[1] 19 💬
385GR181413A/AT1001 capsule----[1] 19 💬
386GR181413A/AT1001 solution----[1] 19 💬
387Growth Hormone----[10] 19, 46, 78, 96, 97, 107, 191, 193, 265, 299 💬
388GSK2788723----[1] 19 💬
389GZ/SAR402671----[2] 19, 67 💬
390GZ385660----[1] 19 💬
391GZ402665----[1] 19 💬
392GZ402671----[1] 19 💬
393GZ402671 / SAR402671----[1] 19 💬
394Hematopoetic Stem Cell Transplantation----[6] 19, 65, 121, 160, 308, 326 💬
395Hematopoietic stem cell infusion----[2] 19, 20 💬
396Hematopoietic Stem Cell Transplantation----[13] 11, 13, 14, 19, 20, 35, 46, 49, 51, 65, 96, 234, 285 💬
397Heparán N sulfatasa humana recombinante----[1] 19 💬
398Heparán N sulfatasa humana recombinante (rhHNS)----[1] 19 💬
399HGT-1110----[1] 19 💬
400HGT-1111----[1] 19 💬
401HGT-1410----[1] 19 💬
402HGT-2310----[1] 19 💬
403HIDS DONOR----[1] 19 💬
404HM15421/GC1134A----[1] 19 💬
405HMI-203[1] HMI-203---[1] 19 💬
406HuCNS-SC----[1] 19 💬
407Human Glucocerebrosidase (prGCD)----[1] 19 💬
408Human N-sulfoglucosamine sulfohydrolase fused to a humanised monoclonal antibody targeting human transferrin receptor----[1] 19 💬
409Human Placental Derived Stem Cell----[4] 19, 20, 60, 284 💬
410Hunterase----[1] 19 💬
411Hydroxypropyl-beta-cyclodextrin----[1] 19 💬
412Hydroxypropyl-ß-cyclodextrin----[1] 19 💬
413Hydroxyurea[1] Hydroxyurea[1] Hydroxyurea 💬 [1] RRM2 💬 [5] Drug metabolism - other enzymes, Glutathione metabolism, Purine metabolism, Pyrimidine metabolism, p53 signaling pathway 💬[10] 3, 13, 19, 20, 46, 65, 85, 86, 107, 284 💬
414I2S----[1] 19 💬
415I2S-IT----[1] 19 💬
416IB1001----[2] 19, 65 💬
417Iduronate-2-sulfatase enzyme replacement therapy----[1] 19 💬
418IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR----[1] 19 💬
419IDURSULFASA[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
420IDURSULFASE[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
421Idursulfase (ELAPRASE)[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
422Idursulfase (I2S)[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
423Idursulfase (I2S)-IT[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
424Idursulfase beta[2] Idursulfase,
Idursulfase beta		[2] Idursulfase ,
Idursulfase beta 💬 		[1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
425Idursulfase IT (1 mg)[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
426Idursulfase IT (10 mg)[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
427Idursulfase IT (30 mg)[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
428Idursulfase(12S)-IT[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
429Idursulfase(I2S)-IT[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
430Idursulfase-IT[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
431IDURSULFASI----[1] 19 💬
432IMD Preparative Regimen----[5] 19, 20, 125, 234, 326 💬
433IMIGLUCERASA----[1] 19 💬
434IMIGLUCERASE[1] Imiglucerase[2] Alglucerase ,
Imiglucerase 💬 		[1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
435Imiglucerase GZ437843[1] Imiglucerase[2] Alglucerase ,
Imiglucerase 💬 		[1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
436IMIGLUCERASI----[1] 19 💬
437Imiglucérase (drug) pharmacokinetics----[1] 19 💬
438Inactive Reminder Capsules----[1] 19 💬
439Inactive Reminder Product----[1] 19 💬
440INN Not available----[2] 19, 46 💬
441INNOZIDE----[1] 19 💬
442Intrathecal recombinant human alpha iduronidase----[1] 19 💬
443Intravenous immunoglobulin (IVIg)----[8] 14, 17, 19, 63, 65, 85, 164, 296 💬
444Intraventricular Access Device----[1] 19 💬
445Iohexol[1] Iohexol[1] Iohexol 💬 --[2] 19, 67 💬
446IRT Laronidase[1] Laronidase[1] Laronidase 💬 [1] IDUA 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
447Isaralgagene civaparvovec[1] Isaralgagene civaparvovec---[1] 19 💬
448Isofagamine tartrate----[1] 19 💬
449ISU302----[1] 19 💬
450IV migalastat HCl[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
451IVA 336----[1] 19 💬
452IZCARGO----[1] 19 💬
453JR-141----[1] 19 💬
454JR-141 or Idursulfase[1] Idursulfase[1] Idursulfase 💬 [1] IDS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
455JR-171----[1] 19 💬
456JR-171 (lepunafusp alfa)[1] Lepunafusp alfa---[1] 19 💬
457JR-441----[1] 19 💬
458JR-446----[1] 19 💬
459KANUMA----[1] 19 💬
460Kidney Transplant----[2] 19, 222 💬
461LAMAZYM[1] Velmanase alfa[1] Velmanase alfa 💬 [1] MAN2B1 💬 [2] Lysosome biogenesis, Other glycan degradation 💬[1] 19 💬
462LARONIDASE[1] Laronidase[1] Laronidase 💬 [1] IDUA 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[2] 19, 256 💬
463Laronidase ERT[1] Laronidase[1] Laronidase 💬 [1] IDUA 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
464Laronidase therapy and a stem cell transplant[1] Laronidase[1] Laronidase 💬 [1] IDUA 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
465Ledipasvir[1] Ledipasvir[1] Ledipasvir 💬 --[1] 19 💬
466Ledipasvir/Sofosbuvir[2] Ledipasvir,
Sofosbuvir		[2] Sofosbuvir ,
Ledipasvir 💬 		--[1] 19 💬
467Lenograstim[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 💬[8] 2, 13, 16, 18, 19, 57, 65, 97 💬
468Lentivirus Alpha-gal A transduced stem cells----[1] 19 💬
469Lepunafusp alfa[1] Lepunafusp alfa---[1] 19 💬
470Leucine[1] Leucine[1] Leucine 💬 --[13] 5, 6, 7, 18, 19, 65, 96, 97, 127, 280, 283, 284, 299 💬
471Leucovorin[1] Leucovorin[3] Leucovorin calcium ,
Folinic acid ,
Folinic acid calcium salt pentahydrate 💬 		--[3] 13, 19, 46 💬
472Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA)----[1] 19 💬
473Lisinopril[1] Lisinopril[2] Lisinopril ,
Lisinopril 💬 		[1] ACE 💬 [6] Chagas disease, Coronavirus disease, Diabetic cardiomyopathy, Hypertrophic cardiomyopathy, Renin secretion, Renin-angiotensin system 💬[9] 19, 46, 49, 57, 66, 67, 113, 222, 223 💬
474Lopace----[1] 19 💬
475Loratadine[1] Loratadine[1] Loratadine 💬 [1] HRH1 💬 [4] Calcium signaling pathway, Inflammatory mediator regulation of TRP channels, Neuroactive ligand signaling, Neuroactive ligand-receptor interaction 💬[5] 13, 19, 46, 89, 98 💬
476Losartan[1] Losartan[2] Losartan potassium ,
Losartan 💬 		[1] AGTR1 💬 [17] AGE-RAGE signaling pathway in diabetic complications, Adrenergic signaling in cardiomyocytes, Aldosterone synthesis and secretion, Apelin signaling pathway, Calcium signaling pathway, Coronavirus disease, Cortisol synthesis and secretion, Cushing syndrome, Diabetic cardiomyopathy, Hormone signaling, Neuroactive ligand-receptor interaction, Pathways in cancer, Phospholipase D signaling pathway, Renin secretion, Renin-angiotensin system, Vascular smooth muscle contraction, cGMP-PKG signaling pathway 💬[20] 17, 19, 36, 46, 57, 58, 66, 67, 85, 96, 97, 98, 113, 164, 167, 179, 215, 222, 223, 299 💬
477Losartan and other angiotensin receptor blockers[1] Losartan[2] Losartan potassium ,
Losartan 💬 		[1] AGTR1 💬 [17] AGE-RAGE signaling pathway in diabetic complications, Adrenergic signaling in cardiomyocytes, Aldosterone synthesis and secretion, Apelin signaling pathway, Calcium signaling pathway, Coronavirus disease, Cortisol synthesis and secretion, Cushing syndrome, Diabetic cardiomyopathy, Hormone signaling, 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 💬
478Lucerastat[1] Lucerastat[1] Lucerastat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
479Lumbar puncture----[9] 2, 6, 13, 14, 19, 96, 97, 127, 201 💬
480LY-M001 Injection----[1] 19 💬
481LY3884961[1] LY3884961---[2] 6, 19 💬
482LYS-GM101----[1] 19 💬
483LYS-SAF302[1] Olenasufligene relduparvovec---[1] 19 💬
484Lysodase----[1] 19 💬
485Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬 --[1] 19 💬
486Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬 --[1] 19 💬
487MABTHERA - 2 FIALE 100 MG 10 ML----[2] 19, 65 💬
488Magnetic Resonance Imaging----[10] 6, 13, 19, 22, 28, 34, 65, 205, 285, 299 💬
489Mechanical stimulation with Von Frey filaments----[1] 19 💬
490Medication to suppress the immune system----[1] 19 💬
491Melphalan[1] Melphalan[2] Melphalan ,
Melphalan hydrochloride 💬 		--[19] 11, 13, 14, 19, 20, 28, 34, 46, 60, 62, 65, 96, 107, 164, 222, 234, 284, 285, 299 💬
492Mepsevii----[1] 19 💬
493Mercaptamina----[1] 19 💬
494Mercaptamine[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
495MERCAPTAMINE BITARTRATE[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
496MERCAPTAMINE HYDROCHLORIDE[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
497Mercaptamine, mercamine, 2-aminoethanethiol[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
498Metazym----[1] 19 💬
499Methotrexate[1] Methotrexate[2] Methotrexate ,
Methotrexate sodium 💬 		[2] DHFR,
DHFR2 💬 		[3] Folate biosynthesis, Folate transport and metabolism, One carbon pool by folate 💬[41] 11, 13, 14, 19, 34, 35, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 53, 54, 55, 56, 60, 62, 65, 84, 93, 96, 97, 107, 162, 164, 172, 223, 240, 256, 270, 271, 284, 285, 286, 326, 331 💬
500Methylprednisolone[1] Methylprednisolone[2] Methylprednisolone ,
Methylprednisolone acetate 💬 		[1] NR3C1 💬 [1] Neuroactive ligand-receptor interaction 💬[51] 2, 6, 11, 13, 14, 18, 19, 25, 26, 35, 38, 39, 40, 41, 42, 43, 44, 45, 46, 49, 50, 51, 60, 62, 63, 64, 65, 66, 83, 84, 85, 86, 93, 95, 96, 97, 111, 127, 145, 162, 164, 211, 222, 223, 224, 271, 283, 284, 285, 296, 300 💬
501MIGALASTAT[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
502Migalastat HCl[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
503Migalastat HCl 150 mg[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
504Migalastat HCl 20 mg[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
505MIGALASTAT HYDROCHLORIDE[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
506Migalastat hydrocloride[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
507MIGLUSTAT[1] Miglustat[1] Miglustat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[3] 19, 256, 299 💬
508MIGLUSTAT (ZAVESCA)[1] Miglustat[1] Miglustat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
509Montelukast[1] Montelukast[2] Montelukast sodium ,
Montelukast 💬 		[1] CYSLTR1 💬 [2] Calcium signaling pathway, Neuroactive ligand-receptor interaction 💬[10] 6, 13, 19, 46, 61, 63, 85, 98, 222, 228 💬
510Moss-aGal (recombinant human alpha-galactosidase A produced in moss)----[1] 19 💬
511MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F----[1] 19 💬
512MRI with contrast agent injection----[1] 19 💬
513MRI without contrast agent injection----[1] 19 💬
514Mucoangin----[1] 19 💬
515Mycophenolate mofetil[2] Mycophenolate mofetil,
Mycophenolic acid		[4] Mycophenolate mofetil ,
Mycophenolate mofetil hydrochloride ,
Mycophenolate sodium ,
Mycophenolic acid 💬 		[2] IMPDH1,
IMPDH2 💬 		[2] Drug metabolism - other enzymes, Purine metabolism 💬[38] 2, 11, 13, 19, 20, 28, 35, 36, 42, 43, 44, 45, 46, 49, 51, 53, 57, 60, 61, 62, 63, 65, 66, 93, 95, 96, 118, 162, 164, 222, 223, 224, 226, 234, 283, 284, 285, 300 💬
516Mycophenylate mofetil----[4] 19, 20, 222, 223 💬
517MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML----[2] 19, 65 💬
518N acetyl cysteine[1] Cysteine[2] L-Cysteine ,
Cysteine hydrochloride 💬 		--[2] 6, 19 💬
519N Acetyl L Leucine[1] Leucine[1] Leucine 💬 --[1] 19 💬
520N-Acetyl-L Leucine[1] Leucine[1] Leucine 💬 --[1] 19 💬
521N-Acetyl-L-Leucine[1] Leucine[1] Leucine 💬 --[3] 18, 19, 65 💬
522N-Acetylcysteine[1] Acetylcysteine[1] Acetylcysteine 💬 --[14] 6, 13, 19, 20, 21, 49, 58, 85, 90, 111, 164, 296, 299, 337 💬
523N-Acetylcysteine Amide[2] Acetylcysteine,
Acetylcysteine amide		[1] Acetylcysteine 💬 --[3] 19, 90, 303 💬
524N-acetylgalactosamine 4-sulfatase----[1] 19 💬
525Naglazyme----[1] 19 💬
526Naglazyme (galsulfase)[1] Galsulfase[1] Galsulfase 💬 [1] ARSB 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
527Naglazyme®----[1] 19 💬
528NGN-101[1] NGN-101---[1] 19 💬
529Nizubaglustat[1] Nizubaglustat[1] Nizubaglustat 💬 [2] GBA2,
UGCG 💬 		[3] Cornified envelope formation, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
530Non applicable----[2] 10, 19 💬
531Odiparcil[1] Odiparcil---[1] 19 💬
532OGT 918----[2] 19, 299 💬
533OGT918----[1] 19 💬
534OGT923----[1] 19 💬
535OLENASUFLIGENE RELDUPARVOVEC[1] Olenasufligene relduparvovec---[1] 19 💬
536OLIPUDASE ALFA[1] Olipudase alfa[1] Olipudase alfa 💬 [1] SMPD1 💬 [4] Lysosome biogenesis, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19 💬
537Olipudase alfa (GZ402665)[1] Olipudase alfa[1] Olipudase alfa 💬 [1] SMPD1 💬 [4] Lysosome biogenesis, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19 💬
538Olipudase alfa (rhASM)[1] Olipudase alfa[1] Olipudase alfa 💬 [1] SMPD1 💬 [4] Lysosome biogenesis, Necroptosis, Sphingolipid metabolism, Sphingolipid signaling pathway 💬[1] 19 💬
539OMNIPAQUE----[2] 19, 222 💬
540Ophthalmology exam----[1] 19 💬
541Optimized Prophylactic IM Therapy----[1] 19 💬
542Oral migalastat HCl[1] Migalastat[2] Migalastat hydrochloride ,
Migalastat 💬 		[1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
543OSOME----[1] 19 💬
544Osteopetrosis Haploidentical Only Preparative Regimen----[5] 19, 20, 125, 234, 326 💬
545Osteopetrosis Only Preparative Regimen----[5] 19, 20, 125, 234, 326 💬
546Other: Blood sampling----[14] 6, 13, 19, 46, 49, 51, 53, 58, 67, 127, 222, 266, 296, 299 💬
547Other: Blood test----[11] 10, 19, 46, 51, 57, 65, 66, 83, 86, 96, 113 💬
548Other: Bone marrow and blood sampling----[1] 19 💬
549Other: Brief Pain Inventory questionnaire----[1] 19 💬
550Other: Challenge agent: capsaicin[1] Capsaicin[1] Capsaicin 💬 [1] TRPV1 💬 [2] Inflammatory mediator regulation of TRP channels, Neuroactive ligand-receptor interaction 💬[1] 19 💬
551Other: Control----[12] 6, 19, 67, 70, 75, 85, 96, 97, 162, 193, 226, 296 💬
552Other: Cysteamine bitartrate[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
553Other: Dried blood spot test----[1] 19 💬
554Other: Dried blood spot test for MPS----[1] 19 💬
555Other: Electroencephalogram (EEG) awake and extended overnight----[1] 19 💬
556Other: extraction of blood samples----[1] 19 💬
557Other: Fluorescein angiography[1] Fluorescein[2] Fluorescein ,
Fluorescein sodium 💬 		--[1] 19 💬
558Other: General and Neurological examination----[1] 19 💬
559Other: Ketogenic Diet----[2] 19, 67 💬
560Other: micromethod from samples taken from blood spots on filter paper----[1] 19 💬
561Other: Neurology exam----[1] 19 💬
562Other: No Investigational Product----[1] 19 💬
563Other: No IT treatment----[1] 19 💬
564Other: Palliative Care----[6] 19, 65, 121, 160, 308, 326 💬
565Other: Potable water[1] Water[1] Water 💬 --[1] 19 💬
566Other: Quality of Life questionnaire----[1] 19 💬
567Other: Sample blood and 2D-echocardiography with Doppler----[2] 19, 58 💬
568Other: Vital signs----[1] 19 💬
569OTL-200----[1] 19 💬
570OTL-200 Dispersion for Infusion----[1] 19 💬
571OTL-203[1] OTL-203---[1] 19 💬
572Oxygen[1] Oxygen[1] Oxygen 💬 --[30] 6, 13, 15, 19, 22, 36, 46, 49, 70, 71, 85, 86, 88, 90, 96, 97, 129, 149, 168, 193, 211, 226, 230, 235, 269, 271, 294, 298, 299, 330 💬
573Oxygen flow at the optic nerve head measurement[1] Oxygen[1] Oxygen 💬 --[1] 19 💬
574Pabinafusp alfa[1] Pabinafusp alfa[1] Pabinafusp alfa 💬 [2] IDS,
TFRC 💬 		[8] Endocytosis, Ferroptosis, Glycosaminoglycan degradation, HIF-1 signaling pathway, Hematopoietic cell lineage, Lysosome biogenesis, TGF-beta signaling pathway, Virion - Lassa virus and SFTS virus 💬[1] 19 💬
575PARACETAMOLO----[10] 2, 11, 14, 19, 28, 53, 61, 63, 300, 309 💬
576PARACETAMOLO DOC GENERICI - 1000 MG COMPRESSA 16 COMPRESSE----[1] 19 💬
577Paricalcitol[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 💬
578PBGM01[1] PBGM01---[1] 19 💬
579PBKR03[1] PBKR03---[1] 19 💬
580PEGUNIGALSIDASE ALFA[1] Pegunigalsidase alfa[1] Pegunigalsidase alfa 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
581Pegunigalsidase alpha----[1] 19 💬
582Pegunigalsidase-alfa----[1] 19 💬
583Pending----[7] 6, 13, 19, 96, 97, 113, 302 💬
584Pentosan Polysulfat SP 54® injection solution----[1] 19 💬
585Pentosan polysulfate[1] Pentosan polysulfate[1] Pentosan polysulfate sodium 💬 --[3] 19, 26, 226 💬
586PENTOSAN POLYSULFATE SODIUM[1] Pentosan polysulfate[1] Pentosan polysulfate sodium 💬 --[1] 19 💬
587Pentosan Polysulphate SP54[1] Pentosan polysulfate[1] Pentosan polysulfate sodium 💬 --[1] 19 💬
588Pentosan Polysulphate SP54®[1] Pentosan polysulfate[1] Pentosan polysulfate sodium 💬 --[1] 19 💬
589Perdix----[1] 19 💬
590Plant cell expressed recombinant glucocerebrosidase (prGCD)----[1] 19 💬
591Plant Cell Recombinant Glucocerebrosidase----[1] 19 💬
592PLX-200----[1] 19 💬
593Prednisone[1] Prednisone[1] Prednisone 💬 [1] NR3C1 💬 [1] Neuroactive ligand-receptor interaction 💬[58] 2, 11, 13, 14, 18, 19, 26, 28, 35, 40, 41, 42, 43, 44, 45, 46, 48, 49, 50, 51, 53, 60, 61, 63, 64, 65, 66, 79, 81, 84, 85, 86, 90, 93, 95, 96, 97, 113, 127, 145, 162, 164, 205, 222, 223, 224, 228, 251, 269, 283, 284, 285, 288, 299, 300, 303, 307, 331 💬
594PREDNISONE 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 💬
595Prevenar13----[3] 19, 79, 265 💬
596PrGCD[1] Taliglucerase alfa[1] Taliglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
597PROCYSBI----[1] 19 💬
598Prophylactic Immunomodulatory (IM) Therapy----[1] 19 💬
599PRX-102----[1] 19 💬
600PRX-102 (pegunigalsidase alfa)[1] Pegunigalsidase alfa[1] Pegunigalsidase alfa 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
601PRX-102 1 mg/kg every 2 weeks----[1] 19 💬
602PRX-102 1 mg/kg every two weeks----[1] 19 💬
603PRX-102 2 mg/kg every 4 weeks----[1] 19 💬
604PRX-112----[1] 19 💬
605PRX102----[1] 19 💬
606PTC124----[4] 19, 113, 246, 299 💬
607Purified form of the lysosomal enzyme iduronate-2-sulfatase----[1] 19 💬
608PYRIMETHAMINE[1] Pyrimethamine[1] Pyrimethamine 💬 --[4] 2, 4, 19, 65 💬
609Quantitative chemical shift imaging (QCSI)----[1] 19 💬
610RAAV 2/6 hIDS DONOR----[1] 19 💬
611RAAV2/5-hNAGLU----[1] 19 💬
612RAAV2/6 Left ZFN Vector----[1] 19 💬
613RAAV2/6 Right ZFN Vector----[1] 19 💬
614RAAV9.CMV.hNAGLU----[1] 19 💬
615Radiation: Total Body Irradiation----[11] 19, 36, 49, 51, 60, 62, 65, 96, 222, 283, 284 💬
616Radiation: Total Body Irradiation with Marrow Boosting----[2] 19, 234 💬
617Rebisufligene etisparvovec[1] Rebisufligene etisparvovec---[1] 19 💬
618Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A----[1] 19 💬
619Recombinant alpha-galactosidase A----[1] 19 💬
620Recombinant human acid sphingomyelinase----[1] 19 💬
621RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)----[1] 19 💬
622Recombinant human alpha galactosidase A (agalsidase beta)[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
623Recombinant human alpha-galactosidase----[1] 19 💬
624Recombinant human alpha-galactosidase A (agalsidase beta)[1] Agalsidase beta[1] Agalsidase beta 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
625Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL----[1] 19 💬
626Recombinant human alpha-mannosidase----[1] 19 💬
627Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)----[1] 19 💬
628RECOMBINANT HUMAN ARYLSULFATASE A[1] Recombinant human arylsulfatase A---[1] 19 💬
629Recombinant Human Arylsulfatase A (rhASA)[1] Recombinant human arylsulfatase A---[1] 19 💬
630Recombinant Human Arylsulfatase A (rhASA,[1] Recombinant human arylsulfatase A---[1] 19 💬
631Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase----[1] 19 💬
632RECOMBINANT HUMAN BETA GLUCURONIDASE----[1] 19 💬
633Recombinant human beta-glucuronidase----[1] 19 💬
634Recombinant human beta-glucuronidase (rhGUS)----[1] 19 💬
635Recombinant human betaglucuronidase----[1] 19 💬
636Recombinant human derived macrophage-targeted beta-Glucocerebrosidase----[1] 19 💬
637Recombinant human derived macrophage-targeted ß-Glucocerebrosidase----[1] 19 💬
638Recombinant human glucerebrosidase----[1] 19 💬
639Recombinant human glucocerebrosidase----[1] 19 💬
640Recombinant Human Glucocerebrosidase (prGCD)[1] Taliglucerase alfa[1] Taliglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
641Recombinant human heparan N-sulfatase----[1] 19 💬
642Recombinant human heparan N-sulfatase (rhHNS)----[1] 19 💬
643Recombinant human heparan N-sulfatase [rhHNS]----[1] 19 💬
644Recombinant human heparan Nsulfatase (rhHNS)----[1] 19 💬
645Recombinant human lysosomal acid lipase----[1] 19 💬
646Recombinant human lysosomal acid lipase (rhLAL)----[1] 19 💬
647Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬 --[1] 19 💬
648Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA)[1] Cholesterol[1] Cholesterol 💬 --[1] 19 💬
649Recombinant human lysosomal alpha-mannosidase----[1] 19 💬
650Recombinant human N-acetylgalactosamine 4-sulfatase----[1] 19 💬
651Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110----[1] 19 💬
652Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance----[1] 19 💬
653Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)----[1] 19 💬
654Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110----[1] 19 💬
655Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance----[1] 19 💬
656Recombinant human Nacetylgalactosamine-----[1] 19 💬
657Recombinant human tripeptidyl peptidase-1[1] Cerliponase alfa[1] Cerliponase alfa 💬 [1] TPP1 💬 [1] Lysosome biogenesis 💬[1] 19 💬
658RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)[1] Cerliponase alfa[1] Cerliponase alfa 💬 [1] TPP1 💬 [1] Lysosome biogenesis 💬[1] 19 💬
659Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene----[1] 19 💬
660Reduced Intensity Conditioning----[2] 19, 65 💬
661Reminder capsules----[1] 19 💬
662Reminder Product----[1] 19 💬
663REPLAGAL----[1] 19 💬
664Replagal (Agalsidase Alfa)[1] Agalsidase alfa[1] Agalsidase alfa 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
665Replagal agalsidase alfa[1] Agalsidase alfa[1] Agalsidase alfa 💬 [1] GLA 💬 [5] Galactose metabolism, Glycerolipid metabolism, Glycosphingolipid biosynthesis - globo and isoglobo series, Lysosome biogenesis, Sphingolipid metabolism 💬[1] 19 💬
666REPLAGAL ®----[1] 19 💬
667RGX-111[1] RGX-111---[1] 19 💬
668RGX-121[1] RGX-121---[1] 19 💬
669Rh Growth Hormone----[1] 19 💬
670RhARSA----[1] 19 💬
671RhASA----[1] 19 💬
672RhASA - Dose Level 1----[1] 19 💬
673RhASA - Dose Level 2----[1] 19 💬
674RhASA - Dose Level 3----[1] 19 💬
675RhASA [recombinant human arylsulfatase A], formerly HGT-1110, now also known as TAK-611[2] Recombinant human arylsulfatase A,
TAK-611		---[1] 19 💬
676RhASM----[1] 19 💬
677RHGUS----[1] 19 💬
678RhHNS-10 mg----[1] 19 💬
679RhHNS-45 mg----[1] 19 💬
680RhHNS-90 mg----[1] 19 💬
681RhIDU (recombinant human-Alpha-L-Iduronidase)----[1] 19 💬
682RhLAMAN[1] Velmanase alfa[1] Velmanase alfa 💬 [1] MAN2B1 💬 [2] Lysosome biogenesis, Other glycan degradation 💬[1] 19 💬
683RhNAGLU----[1] 19 💬
684RhNAGLU radiolabelled----[1] 19 💬
685RHNAGLU-IGF2[1] Tralesinidase alfa[1] Tralesinidase alfa 💬 [2] IGF2,
NAGLU 💬 		[9] Glycosaminoglycan degradation, Hepatocellular carcinoma, Hormone signaling, Lysosome biogenesis, MAPK signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer, Proteoglycans in cancer, Ras signaling pathway 💬[1] 19 💬
686Rimiducid[1] Rimiducid[1] Rimiducid 💬 [1] CASP9 💬 [34] 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, Prion disease, Prostate cancer, Small cell lung cancer, Thyroid hormone signaling pathway, Toxoplasmosis, Tuberculosis, VEGF signaling pathway, Viral myocarditis, p53 signaling pathway 💬[7] 19, 49, 60, 65, 284, 285, 326 💬
687RITUXIMAB[1] Rituximab[1] Rituximab 💬 [1] MS4A1 💬 [1] Hematopoietic cell lineage 💬[54] 11, 13, 14, 19, 26, 34, 35, 36, 40, 41, 42, 43, 44, 45, 46, 48, 49, 50, 51, 52, 53, 55, 56, 60, 61, 62, 63, 64, 65, 66, 83, 84, 85, 86, 88, 93, 94, 96, 97, 162, 172, 222, 223, 229, 256, 271, 274, 283, 284, 285, 288, 296, 300, 331 💬
688Rivogenlecleucel[1] Rivogenlecleucel[1] Rivogenlecleucel 💬 --[6] 19, 60, 65, 284, 285, 326 💬
689RP 103----[2] 8, 19 💬
690RP103----[2] 8, 19 💬
691RVX000222----[1] 19 💬
692SAF-301----[1] 19 💬
693Saline Solution for Injection----[1] 19 💬
694SAR402671, GZ402671 or GZ / SAR402671----[1] 19 💬
695SAR402671, GZ402671 or GZ/SAR402671----[3] 6, 19, 67 💬
696SB-318----[1] 19 💬
697SB-47171----[1] 19 💬
698SB-47898----[1] 19 💬
699SB-913----[1] 19 💬
700SB-A6P-HRL Donor Vector----[1] 19 💬
701SB-FIX----[1] 19 💬
702SB-IDUA----[1] 19 💬
703SBC-102[1] Sebelipase alfa[1] Sebelipase alfa 💬 [1] LIPA 💬 [4] Cholesterol metabolism, Efferocytosis, Lysosome biogenesis, Steroid biosynthesis 💬[1] 19 💬
704SBC-102, recombinant human lysosomal acid lipase[1] Sebelipase alfa[1] Sebelipase alfa 💬 [1] LIPA 💬 [4] Cholesterol metabolism, Efferocytosis, Lysosome biogenesis, Steroid biosynthesis 💬[1] 19 💬
705SBC-103----[1] 19 💬
706ScAAV9.CB.CLN6----[1] 19 💬
707ScAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)----[1] 19 💬
708ScAAV9.U1A.hSGSH[1] Rebisufligene etisparvovec---[1] 19 💬
709ScAAV9.U1A.SGSH----[1] 19 💬
710Sebelipasa Alfa----[1] 19 💬
711Sebelipase Alfa[1] Sebelipase alfa[1] Sebelipase alfa 💬 [1] LIPA 💬 [4] Cholesterol metabolism, Efferocytosis, Lysosome biogenesis, Steroid biosynthesis 💬[1] 19 💬
712Sebelipase alfa (SBC-102)[1] Sebelipase alfa[1] Sebelipase alfa 💬 [1] LIPA 💬 [4] Cholesterol metabolism, Efferocytosis, Lysosome biogenesis, Steroid biosynthesis 💬[1] 19 💬
713Sebelipase alfa 0.35 mg/kg[1] Sebelipase alfa[1] Sebelipase alfa 💬 [1] LIPA 💬 [4] Cholesterol metabolism, Efferocytosis, Lysosome biogenesis, Steroid biosynthesis 💬[1] 19 💬
714Sebelipase alfa 1 mg/kg[1] Sebelipase alfa[1] Sebelipase alfa 💬 [1] LIPA 💬 [4] Cholesterol metabolism, Efferocytosis, Lysosome biogenesis, Steroid biosynthesis 💬[1] 19 💬
715Sebelipase alfa 3 mg/kg[1] Sebelipase alfa[1] Sebelipase alfa 💬 [1] LIPA 💬 [4] Cholesterol metabolism, Efferocytosis, Lysosome biogenesis, Steroid biosynthesis 💬[1] 19 💬
716SHP611----[1] 19 💬
717SHP675----[1] 19 💬
718Sirolimus[1] Sirolimus[1] Sirolimus 💬 [1] MTOR 💬 [52] AMPK signaling pathway, Acute myeloid leukemia, Adipocytokine signaling pathway, Alzheimer disease, Amyotrophic lateral sclerosis, Apelin signaling pathway, Autophagy - animal, Autophagy - other, Breast cancer, Cellular senescence, Central carbon metabolism in cancer, Chemical carcinogenesis - receptor activation, Choline metabolism in cancer, Colorectal cancer, Diabetic cardiomyopathy, ErbB signaling pathway, Gastric cancer, Glioma, Growth hormone synthesis, secretion and action, HIF-1 signaling pathway, Hepatocellular carcinoma, Herpes simplex virus 1 infection, Human cytomegalovirus infection, Human immunodeficiency virus 1 infection, Human papillomavirus infection, Huntington disease, Insulin resistance, Insulin signaling pathway, Integrated stress response (ISR) signaling pathway, Integrin signaling, JAK-STAT signaling pathway, Kaposi sarcoma-associated herpesvirus infection, Longevity regulating pathway, Longevity regulating pathway - multiple species, Lysosome biogenesis, 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 💬[46] 6, 13, 15, 17, 19, 21, 34, 35, 36, 46, 48, 49, 51, 54, 57, 60, 61, 62, 63, 65, 66, 67, 84, 85, 86, 89, 97, 98, 111, 127, 137, 157, 158, 192, 222, 223, 227, 277, 278, 279, 280, 281, 283, 285, 300, 331 💬
719Skeletal survey----[1] 19 💬
720Skin biopsy----[6] 6, 19, 21, 51, 84, 86 💬
721Slit Lamp assessment and intra-ocular pressure measurement----[1] 19 💬
722SOBI003----[1] 19 💬
723Sofosbuvir[1] Sofosbuvir[1] Sofosbuvir 💬 --[1] 19 💬
724Somatropin (DNA origin)----[1] 19 💬
725Speech and modified barium swallow study[1] Barium---[1] 19 💬
726Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride[1] Cysteamine[3] Cysteamine ,
Cysteamine hydrochloride ,
Cysteamine bitartrate 💬 		--[1] 19 💬
727ST-920----[1] 19 💬
728Stem Cell Transplant----[6] 19, 20, 60, 65, 164, 284 💬
729Stem Cell Transplantation----[14] 13, 19, 20, 46, 48, 51, 60, 62, 65, 90, 125, 164, 234, 326 💬
730SUB195712----[1] 19 💬
731Surgery to implant human CNS stem cells (HuCNS-SC)----[1] 19 💬
732TAK-611[1] TAK-611---[1] 19 💬
733TAK-675----[1] 19 💬
734TALIGLUCERASE ALFA[1] Taliglucerase alfa[1] Taliglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
735Taliglucerase alfa - Recombinant human glucocerebrosidase[1] Taliglucerase alfa[1] Taliglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
736TBD----[5] 19, 86, 90, 276, 299 💬
737Temperature sensitivity measurement with Advanced Thermal Stimulation----[1] 19 💬
738Thiotepa[1] Thiotepa[1] Thiotepa 💬 --[14] 13, 19, 20, 28, 46, 51, 60, 65, 85, 96, 107, 164, 284, 285 💬
739Thiotepa--escalated dose[1] Thiotepa[1] Thiotepa 💬 --[4] 19, 20, 65, 284 💬
740Thiotepa--single daily dose[1] Thiotepa[1] Thiotepa 💬 --[4] 19, 20, 65, 284 💬
741Tividenofusp alfa[1] Tividenofusp alfa[1] Tividenofusp alfa 💬 [2] IDS,
TFRC 💬 		[8] Endocytosis, Ferroptosis, Glycosaminoglycan degradation, HIF-1 signaling pathway, Hematopoietic cell lineage, Lysosome biogenesis, TGF-beta signaling pathway, Virion - Lassa virus and SFTS virus 💬[1] 19 💬
742Total body Irradiation----[6] 19, 20, 51, 60, 65, 285 💬
743Tralesinidase alfa[1] Tralesinidase alfa[1] Tralesinidase alfa 💬 [2] IGF2,
NAGLU 💬 		[9] Glycosaminoglycan degradation, Hepatocellular carcinoma, Hormone signaling, Lysosome biogenesis, MAPK signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer, Proteoglycans in cancer, Ras signaling pathway 💬[1] 19 💬
744Tralesinidase alfa (TA)[1] Tralesinidase alfa[1] Tralesinidase alfa 💬 [2] IGF2,
NAGLU 💬 		[9] Glycosaminoglycan degradation, Hepatocellular carcinoma, Hormone signaling, Lysosome biogenesis, MAPK signaling pathway, PI3K-Akt signaling pathway, Pathways in cancer, Proteoglycans in cancer, Ras signaling pathway 💬[1] 19 💬
745Trappsol Cyclo----[1] 19 💬
746TREOSULFAN[1] Treosulfan[1] Treosulfan 💬 --[6] 13, 19, 62, 65, 284, 286 💬
747Triapin----[1] 19 💬
748Triapin& triapin Mite----[1] 19 💬
749Tropicamide[1] Tropicamide[1] Tropicamide 💬 [5] CHRM1,
CHRM2,
CHRM3,
CHRM4,
CHRM5 💬 		[14] Alzheimer disease, Calcium signaling pathway, Cholinergic synapse, Gastric acid secretion, Insulin secretion, Neuroactive ligand signaling, 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 💬
750TSHA-101[1] TSHA-101---[1] 19 💬
751Unrelated Umbilical Cord Blood Transplant----[1] 19 💬
752UPLYSO----[1] 19 💬
753Urine collection----[1] 19 💬
754USAN: sebelipasa alfa----[1] 19 💬
755USAN: sebelipase alpha----[1] 19 💬
756UX003----[1] 19 💬
757UX111----[1] 19 💬
758Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana----[1] 19 💬
759VELAGLUCERASE ALFA[1] Velaglucerase alfa[1] Velaglucerase alfa 💬 [1] GBA1 💬 [4] Cornified envelope formation, Lysosome biogenesis, Other glycan degradation, Sphingolipid metabolism 💬[1] 19 💬
760Velaglucerasi alfa----[1] 19 💬
761VELMANASE ALFA[1] Velmanase alfa[1] Velmanase alfa 💬 [1] MAN2B1 💬 [2] Lysosome biogenesis, Other glycan degradation 💬[1] 19 💬
762Velmanase Alfa (e.g. Lamazym)[1] Velmanase alfa[1] Velmanase alfa 💬 [1] MAN2B1 💬 [2] Lysosome biogenesis, Other glycan degradation 💬[1] 19 💬
763Venglustat[1] Venglustat[2] Venglustat ,
Venglustat malate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[3] 6, 19, 67 💬
764Venglustat (GZ402671)[1] Venglustat[2] Venglustat ,
Venglustat malate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
765Venglustat GZ402671[1] Venglustat[2] Venglustat ,
Venglustat malate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[2] 19, 67 💬
766Venglustat L-malate[1] Venglustat[2] Venglustat ,
Venglustat malate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
767Venglustat malate[1] Venglustat[2] Venglustat ,
Venglustat malate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[3] 6, 19, 67 💬
768Venglustat malato[1] Venglustat[2] Venglustat ,
Venglustat malate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[2] 19, 67 💬
769VenglustatProduct Code: SAR402671, GZ402671 or GZ / SAR402671Pharmaceutical Form: TabletINN or Proposed INN: Venglustat malateCAS Number: 1629063-78-0Current Sponsor code: GZ402671Other descriptive name: GZ/SAR402671Concentration unit: mg milligram(s)Concentration type: equalConcentration number: 6-Pharmaceutical form of the placebo: TabletRoute of administration of the placebo: Oral useProduct Name: venglustatProduct Code: SAR402671, GZ402671 or GZ / SAR402671Pharmaceutical Form: TabletINN or Proposed INN: Venglustat malateCAS Number: 1629063-78-0Current Sponsor code: GZ402671Other descriptive name: GZ/SAR402671Concentration unit: mg milligram(s)Concentration type: equalConcentration number: 15-Pharmaceutical form of the placebo: TabletRoute of administration of the placebo: Oral use[1] Venglustat[2] Venglustat ,
Venglustat malate 💬 		[1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
770Verenafusp alfa 12 mg/kg----[1] 19 💬
771Verenafusp alfa 6 mg/kg----[1] 19 💬
772Verenafusp alfa 9 mg/kg----[1] 19 💬
773Vestronidase alfa[1] Vestronidase alfa[1] Vestronidase alfa 💬 [1] GUSB 💬 [6] Ascorbate and aldarate metabolism, Drug metabolism - other enzymes, Glycosaminoglycan degradation, Lysosome biogenesis, Pentose and glucuronate interconversions, Porphyrin metabolism 💬[1] 19 💬
774VGN-R08b----[1] 19 💬
775Vimizim® (elosulfase alfa)[1] Elosulfase alfa[1] Elosulfase alfa 💬 [1] GALNS 💬 [2] Glycosaminoglycan degradation, Lysosome biogenesis 💬[1] 19 💬
776Visual field testing----[1] 19 💬
777Vitamin D[1] Vitamin D---[26] 3, 6, 13, 15, 19, 41, 43, 44, 46, 49, 50, 68, 74, 93, 95, 96, 97, 98, 179, 222, 228, 235, 256, 274, 298, 299 💬
778Vitamin E[1] Vitamin E[1] Vitamin E 💬 --[12] 2, 6, 13, 19, 20, 46, 90, 96, 97, 206, 299, 317 💬
779Vosoritide[1] Vosoritide[1] Vosoritide 💬 [1] NPR2 💬 [5] Hormone signaling, Oxytocin signaling pathway, Purine metabolism, Vascular smooth muscle contraction, cGMP-PKG signaling pathway 💬[3] 19, 195, 276 💬
780Vosoritide Injection [Voxzogo][1] Vosoritide[1] Vosoritide 💬 [1] NPR2 💬 [5] Hormone signaling, Oxytocin signaling pathway, Purine metabolism, Vascular smooth muscle contraction, cGMP-PKG signaling pathway 💬[2] 19, 276 💬
781VPRIV----[1] 19 💬
782VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO----[1] 19 💬
783VPRIV 400 Units powder for solution of infusion----[1] 19 💬
784VPRIV ®,----[1] 19 💬
785VPRIV®----[1] 19 💬
786VTS-270----[1] 19 💬
787Warfarin[1] Warfarin[3] Warfarin sodium ,
Warfarin potassium ,
Warfarin 💬 		[2] NQO1,
VKORC1 💬 		[5] Chemical carcinogenesis - reactive oxygen species, Fluid shear stress and atherosclerosis, Hepatocellular carcinoma, Pathways in cancer, Ubiquinone and other terpenoid-quinone biosynthesis 💬[15] 6, 8, 13, 19, 46, 48, 49, 66, 85, 86, 88, 91, 96, 97, 222 💬
788Water[1] Water[1] Water 💬 --[36] 2, 3, 5, 6, 13, 17, 19, 22, 35, 46, 49, 51, 53, 56, 57, 62, 63, 64, 65, 66, 67, 70, 72, 85, 86, 96, 97, 206, 215, 225, 226, 230, 240, 274, 278, 299 💬
789Whole body Protein turnover----[4] 19, 46, 96, 299 💬
790ZAVESCA----[3] 19, 256, 299 💬
791Zavesca (Miglustat)[1] Miglustat[1] Miglustat 💬 [1] UGCG 💬 [2] Cornified envelope formation, Sphingolipid metabolism 💬[1] 19 💬
792ZAVESCA 84CPS 100MG----[1] 19 💬
793Zestoretic 10,20----[1] 19 💬
794ZESTORIC----[1] 19 💬
795Zestril----[3] 19, 67, 222 💬