Sources: 44NIH Clinical Center, 4ClinicalTrials, 32LifeMap Discovery®, 6Cochrane Library, 65UMLS, 43NDF-RT, 13DrugBank, 26HMDB, 9DGIdb, 53PharmGKB, 30IUPHAR, 47Novoseek, 1BitterDBSee all MalaCards sources |
Drugs for Lysosomal Storage Disease (from DrugBank, HMDB, Dgidb, PharmGKB, IUPHAR, NovoSeek, BitterDB): (show top 50)
(show all 200)
| id | | Name | Status | Phase | Clinical Trials | Cas Number | PubChem Id |
|---|
| 1 | | Miglustat | approved | Phase 4, Phase 3, Phase 2, Phase 1 | 21 | 72599-27-0 | 51634 | (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol 1,5-Dideoxy-1,5-N-butylimino-D-glucitol 134282-77-2 72599-27-0 AC1L1BHJ BuDNJ Butyldeoxynojirimycin CHEBI:49779 CHEBI:50381 CHEMBL1029 CID51634 D05032 DB00419 HMS2090N20 LS-116261 Miglustat Miglustat (USAN/INN) Miglustat Hydrochloride Miglustat [USAN] Miglustat, Hydrochloride Miglustatum N-(N-Butyl)deoxynojirimycin N-(n-Butyl)deoxynojirimycin N-(n-butyl)-1,5-dideoxy-1,5-imino-D-glucitol | N-Bu-DNJ N-Butyl deoxynojirimycin N-Butyl-1-deoxynojirimycin N-Butyl-DNJ N-Butyl-deoxynojirimycin N-Butyldeoxynojirimycin N-Butylmoranoline N-butyl-1-deoxynojirimycin NB-DNJ NB-dnj NBV OGT 918 OGT-918 SC 48334 SC-48334 SC48334 UNII-ADN3S497AZ Vevesca Zavesca Zavesca (TN) miglustatum n-Butyl deoxynojirimycin n-Butyl dnj nchembio.81-comp12 nchembio850-comp5 | |
| | 2 | | rituximab | approved | Phase 4, Phase 1 | 1582 | 174722-31-7 | 10201696 | AntiCD20 IDEC-102 IDEC-C2B8 | Ig gamma-1 chain C region MabThera Mabthera Rituxan rituximab |
| | 3 | | Cysteamine | approved, investigational | Phase 4, Phase 3, Phase 1, Phase 2 | 23 | 60-23-1 | 6058 | (2-Mercaptoethyl)amine (Mercaptoethyl)ammonium toluene-p-sulphonate .beta.-Mercaptoethylamine 1-Amino-2-mercaptoethylamine 139720-70-0 156-57-0 (hydrochloride) 16904-32-8 (di-hydrochloride) 2-AMINO-ETHANETHIOL 2-AMINO-ethanethiol 2-Amino-1-ethanethiol 2-Aminoethanethiol 2-Aminoethyl mercaptan 2-Mercaptoethanamine 2-Mercaptoethylamine 2-Mercaptoethylamine, polymer-bound 27761-19-9 (tartrate (1:1)) 2DFDA1F8-7010-4225-8280-AB1C4C43F546 30070_FLUKA 30070_SIGMA 3037-04-5 3037-04-5 (tosylate) 42954-15-4 (hydrobromide) 60-23-1 60-23-1 (Parent) 641022_ALDRICH 93965-19-6 (maleate (1:1)) A0648 AC1L1LPL AC1Q54NL AKOS003793343 Aminoethyl mercaptan Becaptan C-9500 C01678 C2H7NS CASH CCRIS 3083 CHEBI:17141 CHEMBL602 CID6058 CYSTEAMINE Cisteamina Cisteamina [Italian] Cystagon Cystaran Cystavision Cysteamide Cysteamin Cysteamine Cysteamine (USAN) Cysteamine Bitartrate Cysteamine Hydrochloride Cysteamine [USAN:BAN] Cysteamine bitartate Cysteamine hydrochloride Cysteaminium Cysteinamine D03634 DB00847 | Decarboxycysteine DivK1c_006750 EINECS 200-463-0 EINECS 221-235-7 Ethanethiolamine HSDB 7353 KBio1_001694 KBio2_002235 KBio2_004803 KBio2_007371 KBioSS_002235 L 1573 L-1573 LS-65761 Lambraten Lambratene Lopac-M-6500 M9768_ALDRICH M9768_SIGMA MEA MEA (mercaptan) Mecramine Mercamin Mercamine Mercaptamin Mercaptamina Mercaptamina [INN-Spanish] Mercaptamine Mercaptamine (INN) Mercaptaminum Mercaptaminum [INN-Latin] Mercaptoethylamine Merkamin MolPort-001-662-635 NCGC00015691-01 NCGC00015691-04 NCGC00162236-01 NCI60_002000 NSC 647528 NSC647528 Riacon STK315355 SpecPlus_000654 Spectrum_001755 Thioethanolamine UNII-5UX2SD1KE2 WR 347 b-Aminoethanethiol b-Aminoethylthiol b-Mercaptoethylamine beta-Aminoethanethiol beta-Aminoethylthiol beta-MEA beta-Mercaptoethylamine bmse000388 cysteamine bitartrate nchembio.315-comp1 nchembio.316-comp1 β-MEA β-aminoethylthiol | |
| | 4 | | Acetylcysteine | approved | Phase 4, Phase 1, Phase 2 | 293 | 616-91-1 | 12035 | (2R)-2-acetylamino-3-Sulfanylpropanoic acid (R)-2-acetylamino-3-Mercaptopropanoic acid (R)-Mercapturic acid 2-Acetylamino-3-mercapto-propionate 2-Acetylamino-3-mercapto-propionic acid ACC Acetadote Acetilcisteina Acetylcysteine Acetylcysteinum Fluimicil Infantil Fluimucetin Fluimucil Flumucetin | Fluprowit L-Acetylcysteine L-alpha-acetamido-beta-Mercaptopropionic acid Lysox Mercapturic acid Mucolysin Mucomyst N-ACETYL-L-cysteine N-Acety-L-Cysteine N-Acetyl-3-mercaptoalanine N-Acetyl-L-(+)-cysteine N-Acetylcysteine N-acetylcysteine NAC Parvolex Sodium 2-acetamido-3-mercaptopropionate |
| | 5 | | Cyclophosphamide | approved, investigational | Phase 4, Phase 2, Phase 3 | 2643 | 50-18-0, 6055-19-2 | 2907 | (+-)-Cyclophosphamide (-)-Cyclophosphamide (RS)-Cyclophosphamide 1-(bis(2-chloroethyl)amino)-1-oxo-2-aza-5-oxaphosphoridine 1-Bis(2-chloroethyl)amino-1-oxo-2-aza-5-oxaphosphoridin 2-[Bis(2-chloroethylamino)]-tetrahydro-2H-1,3,2-oxazaphosphorine-2-oxide 4-Hydroxy-cyclophosphan-mamophosphatide 50-18-0 60007-95-6 6055-19-2 (monohydrate) 75526-90-8 AC1L1EQQ AI3-26198 ASTA ASTA B518 Asta B 518 B 518 B-518 BRN 0011744 BSPBio_002099 Bis(2-chloroethyl)phosphoramide cyclic propanolamide ester C 0768 C07888 C7H15Cl2N2O2P CB 4564 CB-4564 CCRIS 188 CHEBI:4027 CHEMBL32520 CHEMBL88 CID2907 CP CPA CTX CY Ciclofosfamida Ciclofosfamida [INN-Spanish] Ciclofosfamide Ciclophosphamide Ciclophosphamide [INN] Clafen Claphene Cycloblastin Cyclophosphamid Cyclophosphamide Cyclophosphamide (INN) Cyclophosphamide (TN) Cyclophosphamide (anhydrous form) Cyclophosphamide (anhydrous) Cyclophosphamide Monohydrate Cyclophosphamide Sterile Cyclophosphamide anhydrous Cyclophosphamide, (+-)-Isomer Cyclophosphamides Cyclophosphamidum Cyclophosphamidum [INN-Latin] Cyclophosphan Cyclophosphane Cyclophosphanum Cyclophosphoramide Cyclostin Cyklofosfamid Cyklofosfamid [Czech] Cytophosphan Cytophosphane Cytoxan Cytoxan (TN) Cytoxan Lyoph D,L-Cyclophosphamide D07760 DB00531 | DivK1c_000246 EINECS 200-015-4 EU-0100238 Endoxan Endoxan R Endoxan-Asta Endoxana Endoxanal Endoxane Enduxan Genoxal HMS2090A12 HSDB 3047 Hexadrin IDI1_000246 KBio1_000246 KBio2_001338 KBio2_003906 KBio2_006474 KBio3_001319 KBioGR_000888 KBioSS_001338 LS-1302 LS-99787 Ledoxina Lopac-C-0768 Lopac0_000238 Lyophilized Cytoxan Mitoxan MolPort-001-783-420 N,N-Bis(2-chloroethyl)-1,3,2-oxazaphosphinan-2-amine 2-oxide N,N-Bis(2-chloroethyl)tetrahydro-2H-1,3,2-oxazaphosphorin-2-amine 2-oxide NCGC00015209-01 NCGC00015209-03 NCGC00015209-06 NCGC00091741-02 NCGC00091741-03 NCI-C04900 NCI60_002097 NINDS_000246 NSC 26271 NSC-26271 NSC26271 NSC273033 NSC273034 Neosar Occupation, cyclophosphamide exposure Procytox RCRA waste no. U058 Rcra Waste Number U058 Rcra waste number U058 Revimmune S1217_Selleck SK 20501 SPBio_001071 STK177249 STOCK2S-91217 Semdoxan Sendoxan Senduxan Spectrum2_001146 Spectrum3_000370 Spectrum4_000304 Spectrum5_000795 Spectrum_000858 UNII-6UXW23996M WLN: T6MPOTJ BO BN2G2G Zyklophosphamid Zyklophosphamid [German] bis(2-Chloroethyl)phosphami de cyclic propanolamide bis(2-Chloroethyl)phosphamide cyclic propanolamide ester cyclophosphamide | |
| | 6 | | Methotrexate | approved | Phase 4 | 1444 | 1959-05-2, 59-05-2 | 126941 | 4-amino-10-methylfolic acid 4-amino-N(10)-Methylpteroylglutamic acid Abitrexate Amethopterin Amethopterine Antifolan Arbitrexate Emtexate Folex HDMTX L-Amethopterin Ledertrexate MTX Metatrexan Methopterin Methotextrate | Methotrate Methotrexat Methotrexate Methotrexate Sodium Methotrexatum Methylaminopterin Methylaminopterinum Metoject Metotrexato Mexate Méthotrexate N-Bismethylpteroylglutamic Acid N-[4-[[(2,4-Diamino-6-pteridinyl)methyl]methylamino]benzoyl]-L-glutamic acid Otrexup Rasuvo Rheumatrex Rheumatrex Dose Pack Trexall |
| | 7 | | Bortezomib | approved, investigational, experimental | Phase 4 | 769 | 179324-69-7 | 387447, 93860 | 179324-69-7 AC1L8TUW Bortezomib Bortezomib (JAN/USAN/INN) CHEBI:287372 CHEBI:41143 CHEMBL325041 CID387447 D03150 DB07475 DPBA FT-0082488 I14-3268 LDP-341 LDP341 LPD 341 LPD-341 | MLN341 MolPort-003-845-298 N-[(1R)-1-(DIHYDROXYBORYL)-3-METHYLBUTYL]-N-(PYRAZIN-2-YLCARBONYL)-L-PHENYLALANINAMIDE N-[(1R)-1-(DIHYDROXYBORYL)-3-methylbutyl]-N-(pyrazin-2-ylcarbonyl)-L-phenylalaninamide N-[(1R)-1-(dihydroxyboranyl)-3-methylbutyl]-Nalpha-(pyrazin-2-ylcarbonyl)-L-phenylalaninamide NCI60_029010 NSC-681239 NSC681239 PROSCRIPT BORONIC ACID PS 341 PS-341 Pyz-Phe-boroLeu S1013_Selleck SBB071337 Velcade Velcade (TN) Velcade, MG-341, PS-341, Bortezomib [(1R)-3-methyl-1-[[(2S)-3-phenyl-2-(pyrazine-2-carbonylamino)propanoyl]amino]butyl]boronic acid bortezomib |
| | 8 | | Folic Acid | approved, nutraceutical | Phase 4, Phase 1, Phase 2 | 2924 | 59-30-3 | 6037 | (2S)-2-[[4-[(2-amino-4-oxo-1H-pteridin-6-yl)methylamino]benzoyl]amino]pentanedioic acid 01769_FLUKA 2d0k 33609-88-0 36653-55-1 (mono-potassium salt) 59-30-3 6484-89-5 (mono-hydrochloride salt) AC-11682 AC1L1LNX AI3-26387 AKOS000503224 ARONIS014410 Acfol (Spain) Acide folique Acide folique [INN-French] Acido folico Acido folico [INN-Spanish] Acidum folicum Acidum folicum [INN-Latin] Acifolic Antianemia factor Apo-Folic BIDD:ER0563 BIDD:GT0641 BIF0608 BPBio1_000654 BSPBio_000594 BSPBio_002338 C00504 C20H20N6O6 CAS-59-30-3 CCRIS 666 CHEBI:27470 CHEMBL1622 CID6037 CPD000471860 Cytofol D00070 DB00158 DivK1c_000494 Dosfolat B activ EINECS 200-419-0 F0043 F7876_SIAL F7876_SIGMA F8758_SIGMA F8798_SIAL F8890_SIGMA FOL Facid Factor U Folacid Folacin Folaemin Folaemin [Netherlands] Folan Folasic (Australia) Folate Folbal Folcidin Folcidin (VAN) Folcysteine Foldine Foldine [France] Folettes Foliamin Folic Folic Acid Folic acid Folic acid (JP15/USP/INN) Folic acid (TN) Folic acid [BAN:INN:JAN] Folic acid [INN:BAN:JAN] Folic acid dihydrate Folicet Folicet (TN) Folico Folico (Italy) Folina Folina (Italy) Folipac | Folsaeure Folsan Folsaure Folsav Folvite Folvron Glutamic acid, N-(p-(((2-amino-4-hydroxypyrimido(4,5-b)pyrazin-6-yl)methyl)amino)benzoyl)-, L HMS1921D20 HMS2092N17 HMS501I16 HSDB 2002 IDI1_000494 InChI=1/C19H19N7O6/c20-19-25-15-14(17(30)26-19)23-11(8-22-15)7-21-10-3-1-9(2-4-10)16(29)24-12(18(31)32)5-6-13(27)28/h1-4,8,12,21H,5-7H2,(H,24,29)(H,27,28)(H,31,32)(H3,20,22,25,26,30)/t12-/m0/s Incafolic KBio1_000494 KBio2_001861 KBio2_004429 KBio2_006997 KBio3_001558 KBioGR_002222 KBioSS_001861 Kyselina listova Kyselina listova [Czech] LS-2157 Liver Lactobacillus casei factor MLS001304016 MLS001335861 Millafol Mission prenatal Mittafol MolPort-004-285-551 N-(4-(((2-Amino-1,4-dihydro-4-oxo-6-pteridinyl)methyl)amino)benzo- yl)-L-glutamic acid N-(4-((2-Amino-1,4-dihydro-4-oxo-6-pteridinyl)methyl)amino)benzoyl)-L-glutamic acid N-(4-{[(2-Amino-4-oxo-3,4-dihydropteridin-6-yl)methyl]amino}benzoyl)-L-glutamic acid N-(4-{[(2-amino-4-oxo-1,4-dihydropteridin-6-yl)methyl]amino}benzoyl)-L-glutamic acid N-(p-(((2-Amino-4-hydroxy-6-pteridinyl)methyl)amino)benzoyl)-L-glutamic acid N-Pteroyl-L-glutamic acid N-[(4-{[(2-Amino-4-oxo-1,4-dihydropteridin-6-yl)methyl]amino}phenyl)carbonyl]-L-glutamic acid N-[(4-{[(2-amino-4-oxo-1,4-dihydropteridin-6-yl)methyl]amino}phenyl)carbonyl]-L-glutamic acid N-[4-[[(2-Amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-L-glutamic acid NCGC00016265-01 NCGC00142391-01 NINDS_000494 NSC 3073 Nifolin Nifolin [Denmark] Novofolacid Novofolacid [Canada] PGA PGA (VAN) Prestwick3_000627 Prestwick_230 PteGlu Pteroyl-L-glutamate Pteroyl-L-glutamic acid Pteroyl-L-monoglutamate Pteroyl-L-monoglutamic acid Pteroylglutamate Pteroylglutamic acid Pteroylmonoglutamate Pteroylmonoglutamic acid SAM002264616 SDCCGMLS-0066738.P001 SMP2_000137 SMR000471860 SPBio_001357 SPECTRUM1502020 Serum Folate Level Spectrum2_001459 Spectrum3_000749 Spectrum4_001751 Spectrum5_000602 Spectrum_001381 UNII-935E97BOY8 Usaf cb-13 Vitamin B11 Vitamin B9 Vitamin Bc Vitamin Be Vitamin M bmse000299 folic acid nchembio.108-comp10 |
| | 9 | | Anti-Asthmatic Agents | | Phase 4, Phase 1, Phase 2 | 2796 | | | | 10 | | Respiratory System Agents | | Phase 4, Phase 1, Phase 2 | 3931 | | | | 11 | | Antibodies | | Phase 4, Phase 1, Phase 0 | 4477 | | | | 12 | | Immunoglobulins | | Phase 4, Phase 1, Phase 0 | 4477 | | | | 13 | | Bronchodilator Agents | | Phase 4, Phase 1, Phase 2 | 2377 | | | | 14 | | Anti-Retroviral Agents | | Phase 4, Phase 3, Phase 2, Phase 1 | 2794 | | | | 15 | | Adrenergic Agents | | Phase 4, Phase 1, Phase 2 | 4204 | | | | 16 | | Adrenergic Agonists | | Phase 4, Phase 1, Phase 2 | 2331 | | | | 17 | | Adrenergic beta-2 Receptor Agonists | | Phase 4, Phase 1, Phase 2 | 741 | | | | 18 | | Hypoglycemic Agents | | Phase 4, Phase 3, Phase 2, Phase 1 | 4503 | | | | 19 | | Glycoside Hydrolase Inhibitors | | Phase 4, Phase 3, Phase 2, Phase 1 | 111 | | | | 20 | | Protective Agents | | Phase 4, Phase 1, Phase 2 | 5651 | | | | 21 | | Cardiac Glycosides | | Phase 4, Phase 3, Phase 2, Phase 1 | 128 | | | | 22 | | Albuterol | | Phase 4, Phase 1, Phase 2 | 384 | | | | 23 | | Neurotransmitter Agents | | Phase 4, Phase 1, Phase 2, Phase 0 | 14795 | | | | 24 | | Folic Acid Antagonists | | Phase 4, Phase 1, Phase 2 | 1643 | | | | 25 | | Tocolytic Agents | | Phase 4, Phase 1, Phase 2 | 561 | | | | 26 | | Immunologic Factors | | Phase 4, Phase 2, Phase 3, Phase 1, Phase 0 | 18483 | | | | 27 | | Pharmaceutical Solutions | | Phase 4, Phase 2, Phase 3, Phase 1 | 7004 | | | | 28 | | Dermatologic Agents | | Phase 4, Phase 2, Phase 1, Phase 0 | 4555 | | | | 29 | | Antimetabolites | | Phase 4, Phase 2, Phase 1 | 9454 | | | | 30 | | Antimetabolites, Antineoplastic | | Phase 4, Phase 2, Phase 1 | 5770 | | | | 31 | | Antirheumatic Agents | | Phase 4, Phase 2, Phase 3, Phase 1 | 8496 | | | | 32 | | Immunosuppressive Agents | | Phase 4, Phase 2, Phase 3, Phase 1 | 10422 | | | | 33 | | Nucleic Acid Synthesis Inhibitors | | Phase 4, Phase 2 | 3836 | | | | 34 | | Antiviral Agents | | Phase 4, Phase 3, Phase 2, Phase 1 | 8071 | | | | 35 | | Adrenergic beta-Agonists | | Phase 4, Phase 1, Phase 2 | 1266 | | | | 36 | | Expectorants | | Phase 4, Phase 1, Phase 2 | 337 | | | | 37 | | Antioxidants | | Phase 4, Phase 1, Phase 2 | 2442 | | | | 38 | | Anti-Infective Agents | | Phase 4, Phase 3, Phase 2, Phase 1 | 17220 | | | | 39 | | Vitamin B Complex | | Phase 4, Phase 1, Phase 2 | 2847 | | | | 40 | | Peripheral Nervous System Agents | | Phase 4, Phase 2, Phase 1, Phase 0 | 18510 | | | | 41 | | N-monoacetylcystine | | Phase 4, Phase 1, Phase 2 | 293 | | | | 42 | | Immunoglobulins, Intravenous | | Phase 4 | 211 | | | | 43 | | Coal Tar | | Phase 4 | 165 | | | | 44 | | Rho(D) Immune Globulin | | Phase 4 | 206 | | | | 45 | | Folate | Nutraceutical | Phase 4, Phase 1, Phase 2 | 2924 | | | | 46 | | Vitamin B9 | Nutraceutical | Phase 4, Phase 1, Phase 2 | 2924 | | | | 47 | | Folinic Acid | Nutraceutical | Phase 4, Phase 1, Phase 2 | 2604 | | | | 48 | | cysteine | Nutraceutical | Phase 4, Phase 1, Phase 2 | 150 | | | | 49 | | Fludarabine | approved | Phase 2, Phase 3, Phase 1 | 1059 | 21679-14-1, 75607-67-9 | 30751 | (2R,3S,4S,5R)-2-(6-amino-2-fluoro-9H-purin-9-yl)-5-(hydroxymethyl)oxolane-3,4-diol (2R,3S,4S,5R)-2-(6-amino-2-fluoropurin-9-yl)-5-(hydroxymethyl)oxolane-3,4-diol 2-F-ara-A 2-Fluoro Ara-A 2-Fluoro-9-beta-D-arabinofuranosyladenine 2-Fluoro-ara AMP 2-Fluoroadenine arabinoside 5'-monophosphate 21679-14-1 2F-Ara-AMP 9-beta-Arabinofuranosyl-2-fluoroadenine-5'-phosphate 9-beta-D-Arabinofuranosyl-2-fluoroadenine 9-beta-D-Arabinofuranosyl-2-fluoroadenine 5'-(dihydrogen phosphate) 9-beta-D-Arabinofuranosyl-2-fluoroadenine 5'-monophosphate 9-beta-D-arabinofuranosyl-2-fluoro-9H-purin-6-amine 9H-Purin-6-amine, 9-beta-D-arabinofuranosyl-2-fluoro- (9CI) AC1LCW8I AC1Q51CF C10H12FN5O4 CCRIS 3382 CHEMBL1568 CID657237 CPD000058874 D07966 EINECS 244-525-5 F-Ara-A FAMP FT-0082766 FaraA Fludara | Fludara, Fludarabine Fludarabina Fludarabina [Spanish] Fludarabine Fludarabine (INN) Fludarabine 5'-monophosphate Fludarabine Phosphate Fludarabine [INN] Fludarabine monophosphate Fludarabine phosphate Fludarabinum Fludarabinum [Latin] Fludura Fluradosa Fluradosa (TN) HSDB 6964 I14-4978 LS-15061 MLS000028687 NSC 118218 NSC 118218H NSC-118218 Oforta S1491_Selleck SAM002548956 SMR000058874 SQ Fludarabine UNII-1X9VK9O1SC UNII-P2K93U8740 ZINC04216238 | |
| | 50 | | Benzocaine | approved | Phase 3, Phase 2, Phase 1 | 1684 | 1994-09-7, 94-09-7 | 2337 | (p-(Ethoxycarbonyl)phenylamine 06952_FLUKA 112909_ALDRICH 112909_SIAL 1333-08-0 20/20 Topical Anesthetic 23239-88-5 23239-88-5 (hydrochloride) 4 Aminobenzoic Acid Ethyl Ester 4-(Ethoxycarbonyl)aniline 4-(Ethoxycarbonyl)phenylamine 4-14-00-01129 (Beilstein Handbook Reference) 4-Aminobenzoate 4-Aminobenzoic acid 4-Aminobenzoic acid ethyl ester 4-Aminobenzoic acid, ethyl ester 4-Carbethoxyaniline 4-amino-benzoic acid ethyl ester 4-aminobenzoic acid ethyl ester 71123-91-6 94-09-7 94-09-7 (Parent) A0271 AB00051923 AC1L1DGC AC1Q341A AC1Q64JE AE-562/40377256 AI3-02081 AKOS000119763 AR-1H9065 Acetate, Benzocaine Aethoform Aethylium paraminobenzoicum Alcohol Prep Pads Allegenal-m Alpha-caine Topical Anesthetic Amben ethyl ester Americaine Anaesthan-syngala Anaesthesin Anaesthesinum Anaesthin Anbesol Jr Anbesol Maximum Strength Anbesol Regular Strength Anestenka Anestezin Anestezin [Russian] Anesthesin Anesthesine Anesthetic Anesthone Atopalm Oral Pain Relief BB_SC-0019 BPBio1_001017 BRD-K75466013-001-05-2 BRN 0638434 BSPBio_000923 BSPBio_001908 Baby Anbesol Baby Anestenka Baby Sensigel Baby Sensigel Night Time Formula Baby Teething Oral Pain Reliever Bactimicina For Sore Throat Bencocaine Topical Anesthetic Bensokain Benz O Sthetic Benzoak Benzocaina Benzocaina [INN-Spanish] Benzocaine Benzocaine (USP/INN) Benzocaine Acetate Benzocaine Formate Benzocaine Hydrobromide Benzocaine Hydrochloride Benzocaine Methanesulfonate Benzocaine [INN:BAN] Benzocainum Benzocainum [INN-Latin] Benzodent Benzoic acid, 4-amino-, ethyl ester Benzoic acid, 4-amino-, ethyl ester, hydrochloride Benzoic acid, amino-, ethyl ester Benzoic acid, p-amino-, ethyl ester Boil Ease Boy Butter Desensitizing Lubricant Boy Butter Extreme Desensitizing Lubricant Boy Butter Extreme H2O Desensitizing Lubricant Brace Relief Budpak Baby Teething Oral Pain Reliever Budpak Oral Maximum Strength C07527 CAS-94-09-7 CHEBI:116735 CHEMBL278172 CID2337 Carmex Cold Sore Treatment External Analgesic Caswell No. 430A Cepacol Fizzlers Cepacol Sensations Hydra Cepacol Sensations Sore Throat Hydra citrus splash Cepacol Sensations Warming Certus Sting Relief Prep Pad Chloraseptic Chloraseptic Warming Sore Throat Colgate Orabase Comfortcaine Topical Anesthetic Cvs Fast Acting Baby Teething Cvs Maximum Strength Cvs Maximum Strength Boil Relief Cvs Oral Anesthetic Cvs Pharmacy Instant Toothache Cvs Pharmacy Maximum Strength Cvs Pharmacy Mouth Sore Gel Cvs Pharmacy Nighttime Oral Pain Relief D001566 D00552 DB01086 Dental Relief Dentek Instant Pain Relief Maximum Strength Dermoplast Diet Ayds DivK1c_000932 Dollar General Toothache Relief Dukal Sting Relief Pad E1501_SIGMA EINECS 202-303-5 EPA Pesticide Chemical Code 097001 ETHYL-P-AMINOBENZOATE Ethoform Ethoforme Ethyl 4-aminobenzoate Ethyl 4-aminobenzoate hydrochloride Ethyl 4-aminobenzoic acid Ethyl Aminobenzoate Ethyl PABA Ethyl aminobenzoate Ethyl aminobenzoate (JP15) Ethyl aminobenzoate (VAN) Ethyl aminobenzoic acid Ethyl p-Aminobenzoate Ethyl p-Aminophenylcarboxylate Ethyl p-aminobenzenecarboxylate Ethyl p-aminobenzoate Ethyl p-aminobenzoic acid Ethyl p-aminophenylcarboxylate Ethylester kyseliny p-aminobenzoove Ethylester kyseliny p-aminobenzoove [Czech] Ethylis aminobenzoas Finafta Baby Finafta MultiOral Formate, Benzocaine Gelato Topical Anesthetic Good Neighbor Pain Relief Goodsense Oral Pain Relief Gps Topical Anesthetic Gumnumb Topical Anesthetic HMS1570O05 HMS1920G09 HMS2091M11 HMS502O14 HSDB 7225 Heb Severe Toothache Herbasoul Male Genital Desensitizer Cream | Hurricaine Hurricaine Topical Anesthetic Hurricaine Topical Anesthetic Gel Hurricaine Topical Anesthetic Liquid Hydrobromide, Benzocaine Hydrochloride, Benzocaine I05-0204 IDI1_000932 Identhesin Insect Bite Relief Instant Toothache Pain Relief Intense Cvs Intense Toothache Iodent Maximum Strength Oral Analgesic Ismile Topical Anesthetic Jianze Sting Relief Pads KBio1_000932 KBio2_000474 KBio2_003042 KBio2_005610 KBio3_001408 KBioGR_000658 KBioSS_000474 Kank-a Mouth Pain Keloform Kmart Smart Sense Kroger Toothache Pain Relief LS-35847 Leader Oral Analgesic Little Colds Sore Throat Relief Melt Aways MLS001331704 MLS002153970 Makesense Junior Pain Relief Makesense Pain Relief Male Desensitizer Pleasure Balm Mandelay Male Genital Desensitizer Mark 3 Methanesulfonate, Benzocaine MolPort-000-871-526 Mouth Sore Medication Mouth Sore Relief Applicator Mouth Sore Relief Professional Strength My Fair Baby Instant Pain Relief NCGC00016352-01 NCGC00094598-01 NCGC00094598-02 NINDS_000932 NSC 122792 NSC 41531 NSC41531 NSC4688 Nene Dente Norcain Norcaine Norcainum Opahl Opahl Dye Free Oprea1_750694 Oprea1_827402 Ora-jel Orabase-B Orajel For All Mouth Sores Maximum Strength Orajel For Cold Sores Orajel For Toothache Maximum Strength Orajel Instant Pain Relief Maximum Strength Orajel Instant Pain Relief Regular Strength Orajel Instant Relief For Teething Pain Orajel Instant Relief For Teething Pain Cooling Cucumber Orajel Instant Relief For Teething Pain Daytime Nighttime Twin Pack Orajel Instant Relief For Teething Pain Longer Lasting Orajel Maximum Strength Orajel Nighttime Instant Relief For Teething Pain Longer Lasting Extra Strength Orajel Severe Toothache Maximum Strength Fast-Acting Formula Oral Analgesic Maximum Strength Oral B Instant Pain Relief Oral Gel Maximum Strength Oral Pain Relief Anesthetic Anesthetic Oral Pain Reliever Oralabs Cold Sore Treatment Orthesin Otocain Outgro Pac-dent Top Gel Pain Relief Pain Relief Alcohol Swabs Parathesin Parathesin (TN) Parathesine Physicians Care Sting Relief Pad Pleasure Balm Kama Sutra Preboost Prestwick0_000712 Prestwick1_000712 Prestwick2_000712 Prestwick3_000712 Prestwick_991 Purelife Topical Anesthetic Quality Choice Maximum Strength Oral Pain Relieving Red Cross Oral Pain Relief Rexall Maximum Strength Rite Aid Baby Care Rite Aid Liquid Anesthetic Oral Pain Relief Rite Aid Maximum Strength Rite Aid Oral Pain Reliever Rite Aid Professional Strength SMR000059025 SPBio_000134 SPBio_002844 SPECTRUM1500139 STK043620 Sally Hansen Ouch-relief Numbing Wipes Sally Hansen Ouch-relief Wax Kit Sally Hansen Ouch-relief Wax Strip Kit Sensigel Severe Oral Pain Reliever Sheffield Baby Teething Gel Sheffield Pain Relief Shunga Male Genital Desensitizer Slim Mint Gum Solarcaine Solu H Spectrum2_000117 Spectrum3_000314 Spectrum4_000249 Spectrum5_000860 Spectrum_000074 Sting Relief Medicated Pad Sting Relief Pads Tanac Terrasil Boil Pain Relief Tiger Supply Inc Topical Anesthetic Top Quality Mfg. Topical Anesthetic Topcaine Topcare Maximum Strength Topical Anesthetic Banana Topical Anesthetic Bubble Gum Topical Anesthetic Cherry Topical Anesthetic Mint Topical Anesthetic Pina Colada Topical Anesthetic Raspberry Topical Anesthetic Strawberry UNII-U3RSY48JW5 Ultracare Anesthetic Bubble Gum Ultracare Anesthetic Butter Rum Ultracare Anesthetic Creme de Menthe Ultracare Anesthetic Pina Colada Ultracare Anesthetic Walterberry WLN: ZR DVO2 Walgreens Baby Teething Walgreens Intense Toothache Walgreens Maximum Strength Walgreens Pain Relieving For Boils Walgreens Severe Oral Pain Reliever Walgreens Severe Toothache Relief Winco Foods Maximum Strength Oral Analgesic ZINC12358719 Zilactin-b benzocaine ethylaminobenzoate-4 h-4-abz-oet nchembio.182-comp4 p-(Ethoxycarbonyl)aniline p-Aminobenzoate p-Aminobenzoic acid p-Aminobenzoic acid ethyl ester p-Aminobenzoic acid, ethyl ester p-Aminobenzoic ethyl ester p-Carbethoxyaniline p-Ethoxycarboxylic Aniline p-Ethoxycarboxylic aniline |
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Interventional clinical trials: (show top 50)
(show all 486)
| id | Name | Status | NCT ID | Phase |
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| 1 | Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy | Completed | NCT00607386 | Phase 4 | | 2 | Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease | Completed | NCT00364858 | Phase 4 | | 3 | Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa | Completed | NCT01288027 | Phase 4 | | 4 | Late-Onset Treatment Study Extension Protocol | Completed | NCT00455195 | Phase 4 | | 5 | High Dose or High Dose Frequency Study of Alglucosidase Alfa | Completed | NCT00483379 | Phase 4 | | 6 | An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease | Completed | NCT00701129 | Phase 4 | | 7 | Replagal Enzyme Replacement Therapy for Adults With Fabry Disease | Completed | NCT00097890 | Phase 4 | | 8 | Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. | Completed | NCT01997489 | Phase 4 | | 9 | A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. | Completed | NCT00365131 | Phase 4 | | 10 | A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease | Completed | NCT01132690 | Phase 4 | | 11 | A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease | Completed | NCT00140621 | Phase 4 | | 12 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | Completed | NCT00081497 | Phase 4 | | 13 | A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease | Completed | NCT00074984 | Phase 4 | | 14 | A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. | Completed | NCT00233870 | Phase 4 | | 15 | A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta | Completed | NCT01650779 | Phase 4 | | 16 | N Acetyl Cysteine for Cystinosis Patients | Completed | NCT01614431 | Phase 4 | | 17 | A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease | Completed | NCT00144781 | Phase 4 | | 18 | A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients | Completed | NCT00144768 | Phase 4 | | 19 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | Completed | NCT00299000 | Phase 4 | | 20 | A Study of the Effects of Fabrazyme (Agalsidase Beta) on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants | Recruiting | NCT00230607 | Phase 4 | | 21 | Pompe Pregnancy Sub-Registry | Recruiting | NCT00567073 | Phase 4 | | 22 | Pompe Lactation Sub-Registry | Recruiting | NCT00566878 | Phase 4 | | 23 | Immune Tolerance Induction Study | Recruiting | NCT00701701 | Phase 4 | | 24 | Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8 Years of Age and Older | Recruiting | NCT01410890 | Phase 4 | | 25 | Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study | Recruiting | NCT00455104 | Phase 4 | | 26 | Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease | Recruiting | NCT00487630 | Phase 4 | | 27 | The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease | Recruiting | NCT02528617 | Phase 4 | | 28 | CPAP for Infantile Pompe Disease | Recruiting | NCT02405624 | Phase 4 | | 29 | Immune Modulation Therapy for Pompe Disease | Recruiting | NCT02525172 | Phase 4 | | 30 | Synergistic Enteral Regimen for Treatment of the Gangliosidoses | Recruiting | NCT02030015 | Phase 4 | | 31 | This Study is to Evaluate the Effect of Velaglucerase Alfa (VPRIV®) on Patients With Type 1 Gaucher Disease Through the IV Administration of VPRIV® Over 2 Years | Recruiting | NCT02574286 | Phase 4 | | 32 | Use of Cysteamine in the Treatment of Cystinosis | Recruiting | NCT00359684 | Phase 4 | | 33 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | Recruiting | NCT00214773 | Phase 4 | | 34 | A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants | Recruiting | NCT00418821 | Phase 4 | | 35 | Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age | Recruiting | NCT02455622 | Phase 4 | | 36 | Growth and Development Study of Alglucosidase Alfa. | Active, not recruiting | NCT00486889 | Phase 4 | | 37 | Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease | Active, not recruiting | NCT02405598 | Phase 4 | | 38 | A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease | Terminated | NCT01597596 | Phase 4 | | 39 | A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease | Terminated | NCT01526785 | Phase 4 | | 40 | Morquio's Syndrome: a Case Study | Terminated | NCT00609440 | Phase 4 | | 41 | A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency. | Withdrawn | NCT00312767 | Phase 4 | | 42 | Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase | Completed | NCT00630747 | Phase 2, Phase 3 | | 43 | Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase | Completed | NCT00712348 | Phase 3 | | 44 | A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease | Completed | NCT00376168 | Phase 3 | | 45 | Open-Label Phase 3 Long-Term Safety Study of Migalastat | Completed | NCT01458119 | Phase 3 | | 46 | Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial | Completed | NCT00705939 | Phase 3 | | 47 | Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase | Completed | NCT00478647 | Phase 2, Phase 3 | | 48 | Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease | Completed | NCT00553631 | Phase 3 | | 49 | A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease | Completed | NCT00430625 | Phase 3 | | 50 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Completed | NCT01275066 | Phase 3 |
Cell-based therapeutics:Data from LifeMap, the Embryonic Development and Stem Cells Database Stem-cell-based therapeutic approaches for Lysosomal Storage Disease: Embryonic/Adult Cultured Cells Related to Lysosomal Storage Disease:
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