Drugs for Lysosomal Storage Disease (from DrugBank, HMDB, Dgidb, PharmGKB, IUPHAR, NovoSeek, BitterDB):
(show top 50)
(show all 215)
# |
|
Name |
Status |
Phase |
Clinical Trials |
Cas Number |
PubChem Id |
1 |
|
Cysteamine |
Approved, Investigational |
Phase 4,Phase 3,Phase 2,Phase 1 |
|
60-23-1 |
6058
|
Synonyms:
(2-Mercaptoethyl)amine
(Mercaptoethyl)ammonium toluene-p-sulphonate
.beta.-Mercaptoethylamine
139720-70-0
156-57-0 (hydrochloride)
16904-32-8 (di-hydrochloride)
1-Amino-2-mercaptoethylamine
27761-19-9 (tartrate (1:1))
2-Amino-1-ethanethiol
2-Aminoethanethiol
2-AMINO-ethanethiol
2-AMINO-ETHANETHIOL
2-Aminoethyl mercaptan
2DFDA1F8-7010-4225-8280-AB1C4C43F546
2-Mercaptoethanamine
2-Mercaptoethylamine
2-Mercaptoethylamine, polymer-bound
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
b-Aminoethanethiol
b-Aminoethylthiol
Becaptan
beta-Aminoethanethiol
beta-Aminoethylthiol
beta-MEA
beta-Mercaptoethylamine
b-Mercaptoethylamine
bmse000388
C01678
C2H7NS
C-9500
CASH
CCRIS 3083
CHEBI:17141
CHEMBL602
CID6058
Cisteamina
Cisteamina [Italian]
Cystavision
Cysteamide
Cysteamin
Cysteamine
CYSTEAMINE
Cysteamine (USAN)
Cysteamine [USAN:BAN]
Cysteamine bitartate
|
cysteamine bitartrate
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
Lambraten
Lambratene
Lopac-M-6500
LS-65761
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
nchembio.315-comp1
nchembio.316-comp1
NCI60_002000
NSC 647528
NSC647528
Riacon
SpecPlus_000654
Spectrum_001755
STK315355
Thioethanolamine
UNII-5UX2SD1KE2
WR 347
β-aminoethylthiol
β-MEA
|
|
2 |
|
Cyclophosphamide |
Approved, Investigational |
Phase 4,Phase 2,Phase 3 |
|
50-18-0, 6055-19-2 |
2907
|
Synonyms:
(-)-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
Anhydrous cyclophosphamide
ASTA
Asta B 518
ASTA B518
B 518
B-518
bis(2-Chloroethyl)phosphami de cyclic propanolamide
bis(2-Chloroethyl)phosphamide cyclic propanolamide ester
Bis(2-chloroethyl)phosphoramide cyclic propanolamide ester
BRN 0011744
BSPBio_002099
C 0768
C07888
C7H15Cl2N2O2P
CB 4564
CB-4564
CCRIS 188
CHEBI:4027
CHEMBL32520
CHEMBL88
Ciclofosfamida
Ciclofosfamida [INN-Spanish]
Ciclofosfamide
Ciclophosphamide
Ciclophosphamide [INN]
CID2907
Clafen
Claphene
CP
CPA
CTX
CY
Cycloblastin
Cyclophosphamid
cyclophosphamide
Cyclophosphamide
Cyclophosphamide (anhydrous form)
Cyclophosphamide (anhydrous)
Cyclophosphamide (INN)
Cyclophosphamide (TN)
Cyclophosphamide anhydrous
Cyclophosphamide Monohydrate
Cyclophosphamide Sterile
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
Endoxan
Endoxan R
Endoxana
Endoxanal
Endoxan-Asta
Endoxane
Enduxan
EU-0100238
Genoxal
Hexadrin
HMS2090A12
HSDB 3047
IDI1_000246
KBio1_000246
KBio2_001338
KBio2_003906
KBio2_006474
KBio3_001319
KBioGR_000888
KBioSS_001338
Ledoxina
Lopac0_000238
Lopac-C-0768
LS-1302
LS-99787
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
NCI60_002097
NCI-C04900
Neosar
NINDS_000246
NSC 26271
NSC26271
NSC-26271
NSC273033
NSC273034
Occupation, cyclophosphamide exposure
Procytox
RCRA waste no. U058
Rcra waste number U058
Rcra Waste Number U058
Revimmune
S1217_Selleck
Semdoxan
Sendoxan
Senduxan
SK 20501
SPBio_001071
Spectrum_000858
Spectrum2_001146
Spectrum3_000370
Spectrum4_000304
Spectrum5_000795
STK177249
STOCK2S-91217
UNII-6UXW23996M
WLN: T6MPOTJ BO BN2G2G
Zyklophosphamid
Zyklophosphamid [German]
|
|
3 |
|
Acetylcysteine |
Approved, Investigational |
Phase 4,Phase 1,Phase 2 |
|
616-91-1 |
12035
|
Synonyms:
(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-Acetylcysteine
Lysox
L-α-acetamido-β-mercaptopropionic acid
Mercapturic acid
Mucolysin
Mucomyst
NAC
N-Acetyl-3-mercaptoalanine
N-acetylcysteine
N-Acetylcysteine
N-Acety-L-Cysteine
N-acetyl-L-(+)-cysteine
N-Acetyl-L-(+)-cysteine
N-acetyl-L-cysteine
Parvolex
Sodium 2-acetamido-3-mercaptopropionate
|
|
4 |
|
Methotrexate |
Approved |
Phase 4 |
|
1959-05-2, 59-05-2 |
126941
|
Synonyms:
4-amino-10-methylfolic acid
4-amino-N(10)-methylpteroylglutamic acid
Abitrexate
Amethopterin
Amethopterine
Antifolan
Arbitrexate
Emtexate
Folex
HDMTX
L-Amethopterin
Ledertrexate
Metatrexan
Methopterin
Methotextrate
|
Methotrate
Methotrexat
Méthotrexate
Methotrexate Sodium
Methotrexatum
Methylaminopterin
Methylaminopterinum
Metotrexato
Mexate
MTX
N-[4-[[(2,4-diamino-6-pteridinyl)methyl]methylamino]benzoyl]-L-glutamic acid
N-Bismethylpteroylglutamic Acid
Rheumatrex
Trexall
|
|
5 |
|
rituximab |
Approved |
Phase 4,Phase 2,Phase 1 |
|
174722-31-7 |
10201696
|
Synonyms:
AntiCD20
IDEC-102
IDEC-C2B8
Ig gamma-1 chain C region
|
Mabthera
MabThera
Rituxan
rituximab
|
|
6 |
|
Miglustat |
Approved |
Phase 4,Phase 3,Phase 2,Phase 1 |
|
72599-27-0 |
51634
|
Synonyms:
(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 [USAN]
Miglustat, Hydrochloride
miglustatum
Miglustatum
N-(n-butyl)-1,5-dideoxy-1,5-imino-D-glucitol
N-(n-Butyl)deoxynojirimycin
N-(N-Butyl)deoxynojirimycin
|
NB-dnj
NB-DNJ
N-Bu-DNJ
n-Butyl deoxynojirimycin
N-Butyl deoxynojirimycin
n-Butyl dnj
N-butyl-1-deoxynojirimycin
N-Butyl-1-deoxynojirimycin
N-Butyldeoxynojirimycin
N-Butyl-deoxynojirimycin
N-Butyl-DNJ
N-Butylmoranoline
NBV
nchembio.81-comp12
nchembio850-comp5
OGT 918
OGT-918
SC 48334
SC48334
SC-48334
UNII-ADN3S497AZ
Vevesca
Zavesca
Zavesca (TN)
|
|
7 |
|
Bortezomib |
Approved, Investigational |
Phase 4 |
|
179324-69-7 |
387447
93860
|
Synonyms:
[(1R)-3-methyl-1-({(2S)-3-phenyl-2-[(pyrazin-2-ylcarbonyl)amino]propanoyl}amino)butyl]boronic acid
[(1R)-3-methyl-1-[[(2S)-3-phenyl-2-(pyrazine-2-carbonylamino)propanoyl]amino]butyl]boronic acid
179324-69-7
AC1L8TUW
bortezomib
Bortezomib
Bortezomib (JAN/USAN/INN)
CHEBI:287372
CHEBI:41143
CHEMBL325041
CID387447
D03150
DB07475
DPBA
FT-0082488
I14-3268
LDP341
LDP-341
|
LPD 341
LPD-341
MLN341
MolPort-003-845-298
N-[(1R)-1-(dihydroxyboranyl)-3-methylbutyl]-Nalpha-(pyrazin-2-ylcarbonyl)-L-phenylalaninamide
N-[(1R)-1-(DIHYDROXYBORYL)-3-methylbutyl]-N-(pyrazin-2-ylcarbonyl)-L-phenylalaninamide
N-[(1R)-1-(DIHYDROXYBORYL)-3-METHYLBUTYL]-N-(PYRAZIN-2-YLCARBONYL)-L-PHENYLALANINAMIDE
NCI60_029010
NSC681239
NSC-681239
PROSCRIPT BORONIC ACID
PS-341
Pyz-Phe-boroLeu
S1013_Selleck
SBB071337
Velcade
Velcade (TN)
Velcade, MG-341, PS-341, Bortezomib
|
|
8 |
|
Folic Acid |
Approved, Nutraceutical, Vet_approved |
Phase 4,Phase 1,Phase 2 |
|
59-30-3 |
6037
|
Synonyms:
(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
Acfol (Spain)
Acide folique
Acide folique [INN-French]
Acido folico
Acido folico [INN-Spanish]
Acidum folicum
Acidum folicum [INN-Latin]
Acifolic
AI3-26387
AKOS000503224
Antianemia factor
Apo-Folic
ARONIS014410
BIDD:ER0563
BIDD:GT0641
BIF0608
bmse000299
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
Facid
Factor U
FOL
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
Incafolic
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
KBio1_000494
KBio2_001861
KBio2_004429
KBio2_006997
KBio3_001558
KBioGR_002222
KBioSS_001861
Kyselina listova
Kyselina listova [Czech]
Liver Lactobacillus casei factor
LS-2157
Millafol
Mission prenatal
Mittafol
MLS001304016
MLS001335861
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-1,4-dihydropteridin-6-yl)methyl]amino}benzoyl)-L-glutamic acid
N-(4-{[(2-Amino-4-oxo-3,4-dihydropteridin-6-yl)methyl]amino}benzoyl)-L-glutamic acid
N-(p-(((2-Amino-4-hydroxy-6-pteridinyl)methyl)amino)benzoyl)-L-glutamic acid
N-[(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
nchembio.108-comp10
Nifolin
Nifolin [Denmark]
NINDS_000494
Novofolacid
Novofolacid [Canada]
N-Pteroyl-L-glutamic acid
NSC 3073
PGA
PGA (VAN)
Prestwick_230
Prestwick3_000627
PteGlu
Pteroylglutamate
Pteroylglutamic acid
Pteroyl-L-glutamate
Pteroyl-L-glutamic acid
Pteroyl-L-monoglutamate
Pteroyl-L-monoglutamic acid
Pteroylmonoglutamate
Pteroylmonoglutamic acid
SAM002264616
SDCCGMLS-0066738.P001
Serum Folate Level
SMP2_000137
SMR000471860
SPBio_001357
Spectrum_001381
SPECTRUM1502020
Spectrum2_001459
Spectrum3_000749
Spectrum4_001751
Spectrum5_000602
UNII-935E97BOY8
Usaf cb-13
Vitamin B11
Vitamin B9
Vitamin Bc
Vitamin Be
Vitamin M
|
|
9 |
|
leucovorin |
Approved, Nutraceutical |
Phase 4,Phase 1,Phase 2 |
|
58-05-9 |
143
6006
|
Synonyms:
(5-formyl-5,6,7,8-tetrahydropteroyl)glutamate
(6R,S)-5-Formyltetrahydrofolate
10-Formyl-7,8-dihydrofolate
10-Formyl-7,8-dihydrofolic acid
5-Formyl-5,6,7,8-tetrahydrofolate
5-Formyl-5,6,7,8-tetrahydrofolic acid
5-Formyl-5,6,7,8-tetrahydropteroyl-L-glutamic acid
5-Formyltetrahydrofolate
5-formyltetrahydrofolic acid
5-Formyltetrahydrofolic acid
5-Formyltetrahydropteroylglutamate
5-Formyltetrahydropteroylglutamic acid
Acide folinique
Calcium citrovorum factor
Calcium folinate
Citrovorum factor
folinate
Folinate
Folinic acid
Folinic acid calcium salt
|
Folinic acid calcium salt USP27
Folinic acid-SF
L(-)-5-Formyl-5,6,7,8-tetrahydrofolic acid
Leucal
Leucovorin calcium
Leucovorin folinic acid
Leucovorinum
Leukovorin
Levoleucovorin
L-leucovorin
L-Leucovorin
L-N-[p-[[(2-Amino-5-formyl-5,6,7,8-tetrahydro-4-hydroxy-6-pteridinyl)methyl]amino]benzoyl]-Glutamic acid
N-(5-formyl-5,6,7,8-tetrahydropteroyl)-L-glutamic acid
N5-Formyl-5,6,7,8-tetrahydrofolate
N5-Formyl-5,6,7,8-tetrahydrofolic acid
N5-Formyltetrahydrofolate
N5-Formyltetrahydrofolic acid
N5-Formyl-THF
Welcovorin
Wellcovorin
|
|
10 |
|
Vitamin D |
Approved, Nutraceutical, Vet_approved |
Phase 4,Phase 2 |
|
1406-16-2 |
|
Synonyms:
|
Vitamin D, unspecified form
|
|
11 |
|
1-Deoxynojirimycin |
Experimental |
Phase 4,Phase 3,Phase 2,Phase 1 |
|
19130-96-2 |
1374
|
Synonyms:
(2R,3R,4R,5S)-2-(Hydroxymethyl)piperidine-3,4,5-triol
1 deoxynojirimycin
1,5-deoxy-1,5-imino-D-mannitol
1,5-dideoxy-1,5-imino-D-glucitol
1,5-Dideoxy-1,5-imino-D-glucitol, 9CI
1-Deoxymannojirimycin
1-deoxynojirimycin
1-deoxy-nojirimycin
1-Deoxynojirimycin
1-DEOXY-NOJIRIMYCIN
1-Deoxynojirimycin (DNJ)
|
1hxk
1oim
5-amino-1,5-dideoxy-D-glucopyranose
Antibiotic S-GI
D-1-deoxynojirimycin
Deoxynojirimycin
DNJ
Moranoline
NOJ
S-GI
|
|
12 |
|
Immunosuppressive Agents |
|
Phase 4,Phase 2,Phase 3,Phase 1 |
|
|
|
13 |
|
Pharmaceutical Solutions |
|
Phase 4,Phase 2,Phase 3,Phase 1 |
|
|
|
14 |
|
Antidotes |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
15 |
|
Anti-Infective Agents |
|
Phase 4,Phase 3,Phase 2,Phase 1 |
|
|
|
16 |
|
Antioxidants |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
17 |
|
Antiviral Agents |
|
Phase 4,Phase 3,Phase 2,Phase 1 |
|
|
|
18 |
|
Expectorants |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
19 |
|
N-monoacetylcystine |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
20 |
|
Protective Agents |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
21 |
|
Respiratory System Agents |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
22 |
|
Adrenergic Agents |
|
Phase 4,Phase 2,Phase 1 |
|
|
|
23 |
|
Adrenergic Agonists |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
24 |
|
Adrenergic beta-2 Receptor Agonists |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
25 |
|
Adrenergic beta-Agonists |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
26 |
|
Albuterol |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
27 |
|
Anti-Asthmatic Agents |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
28 |
|
Autonomic Agents |
|
Phase 4,Phase 2,Phase 1 |
|
|
|
29 |
|
Bronchodilator Agents |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
30 |
|
Neurotransmitter Agents |
|
Phase 4,Phase 2,Phase 1,Early Phase 1 |
|
|
|
31 |
|
Peripheral Nervous System Agents |
|
Phase 4,Phase 2,Phase 1,Early Phase 1 |
|
|
|
32 |
|
Tocolytic Agents |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
33 |
|
Antimetabolites |
|
Phase 4,Phase 2,Phase 1 |
|
|
|
34 |
|
Antimetabolites, Antineoplastic |
|
Phase 4,Phase 2,Phase 1 |
|
|
|
35 |
|
Antirheumatic Agents |
|
Phase 4,Phase 2,Phase 3,Phase 1 |
|
|
|
36 |
|
Dermatologic Agents |
|
Phase 4,Phase 2,Phase 1,Early Phase 1 |
|
|
|
37 |
|
Folic Acid Antagonists |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
38 |
|
Nucleic Acid Synthesis Inhibitors |
|
Phase 4,Phase 2 |
|
|
|
39 |
|
Vitamin B Complex |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
40 |
|
Antibodies |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
41 |
|
Immunoglobulins |
|
Phase 4,Phase 1,Phase 2 |
|
|
|
42 |
|
Bone Density Conservation Agents |
|
Phase 4,Phase 2 |
|
|
|
43 |
|
Micronutrients |
|
Phase 4,Phase 2 |
|
|
|
44 |
|
Trace Elements |
|
Phase 4,Phase 2 |
|
|
|
45 |
|
Vitamins |
|
Phase 4,Phase 2 |
|
|
|
46 |
|
Anti-HIV Agents |
|
Phase 4,Phase 3,Phase 2,Phase 1 |
|
|
|
47 |
|
Anti-Retroviral Agents |
|
Phase 4,Phase 3,Phase 2,Phase 1 |
|
|
|
48 |
|
Cardiac Glycosides |
|
Phase 4,Phase 3,Phase 2,Phase 1 |
|
|
|
49 |
|
Glycoside Hydrolase Inhibitors |
|
Phase 4,Phase 3,Phase 2,Phase 1 |
|
|
|
50 |
|
Hypoglycemic Agents |
|
Phase 4,Phase 3,Phase 2,Phase 1 |
|
|
|
Interventional clinical trials:
(show top 50)
(show all 571)
# |
Name |
Status |
NCT ID |
Phase |
Drugs |
1 |
CPAP for Infantile Pompe Disease |
Unknown status |
NCT02405624
|
Phase 4 |
|
2 |
Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease |
Unknown status |
NCT00487630
|
Phase 4 |
recombinant alpha-galactosidase A |
3 |
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy |
Completed |
NCT00607386
|
Phase 4 |
|
4 |
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease |
Completed |
NCT00364858
|
Phase 4 |
Cerezyme |
5 |
N Acetyl Cysteine for Cystinosis Patients |
Completed |
NCT01614431
|
Phase 4 |
N acetyl cysteine;N acetyl cysteine |
6 |
Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease |
Completed |
NCT02405598
|
Phase 4 |
Salbutamol |
7 |
Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. |
Completed |
NCT01997489
|
Phase 4 |
Enzyme replacement |
8 |
A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta |
Completed |
NCT01650779
|
Phase 4 |
|
9 |
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa |
Completed |
NCT01288027
|
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 |
Taliglucerase alfa |
11 |
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 |
Methotrexate;Rituximab |
12 |
High Dose or High Dose Frequency Study of Alglucosidase Alfa |
Completed |
NCT00483379
|
Phase 4 |
|
13 |
Late-Onset Treatment Study Extension Protocol |
Completed |
NCT00455195
|
Phase 4 |
|
14 |
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. |
Completed |
NCT00365131
|
Phase 4 |
Cerezyme (imiglucerase for injection) |
15 |
A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. |
Completed |
NCT00233870
|
Phase 4 |
Agalsidase beta (recombinant form) |
16 |
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease |
Completed |
NCT00140621
|
Phase 4 |
Agalsidase beta |
17 |
Replagal Enzyme Replacement Therapy for Adults With Fabry Disease |
Completed |
NCT00097890
|
Phase 4 |
Replagal (Agalsidase Alfa);Replagal |
18 |
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease |
Completed |
NCT00081497
|
Phase 4 |
|
19 |
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 |
|
20 |
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI |
Completed |
NCT00299000
|
Phase 4 |
Naglazyme |
21 |
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease |
Completed |
NCT00144781
|
Phase 4 |
|
22 |
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients |
Completed |
NCT00144768
|
Phase 4 |
laronidase |
23 |
Study of the Effects of Fabrazyme Treatment on Lactation and Infants |
Recruiting |
NCT00230607
|
Phase 4 |
agalsidase beta |
24 |
Use of Cysteamine in the Treatment of Cystinosis |
Recruiting |
NCT00359684
|
Phase 4 |
Cysteamine |
25 |
Immune Modulation Therapy for Pompe Disease |
Recruiting |
NCT02525172
|
Phase 4 |
Rituximab;intravenous immune globulin;Bortezomib;Methotrexate |
26 |
Phase 4 Study 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 |
Velaglucerase alfa |
27 |
Synergistic Enteral Regimen for Treatment of the Gangliosidoses |
Recruiting |
NCT02030015
|
Phase 4 |
miglustat |
28 |
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease |
Recruiting |
NCT01410890
|
Phase 4 |
|
29 |
Immune Tolerance Induction Study |
Recruiting |
NCT00701701
|
Phase 4 |
|
30 |
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age |
Recruiting |
NCT02455622
|
Phase 4 |
Elaprase for intravenous (IV) infusion |
31 |
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 |
|
32 |
Growth and Development Study of Alglucosidase Alfa. |
Active, not recruiting |
NCT00486889
|
Phase 4 |
|
33 |
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease |
Not yet recruiting |
NCT03021941
|
Phase 4 |
Elelyso 60 units/kg |
34 |
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 |
|
35 |
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease |
Terminated |
NCT01526785
|
Phase 4 |
Alglucosidase alfa |
36 |
Morquio's Syndrome: a Case Study |
Terminated |
NCT00609440
|
Phase 4 |
|
37 |
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease |
Withdrawn |
NCT02528617
|
Phase 4 |
Velaglucerase alfa |
38 |
A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency. |
Withdrawn |
NCT00312767
|
Phase 4 |
Fabrazyme (agalsidase beta) |
39 |
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis |
Unknown status |
NCT01238328
|
Phase 2, Phase 3 |
Conditioning regimen;Graft-versus-host disease (GVHD) prophylaxis |
40 |
BMN 110 Phase 3B in Australian Patients |
Unknown status |
NCT01966029
|
Phase 3 |
BMN 110 |
41 |
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase |
Completed |
NCT00630747
|
Phase 2, Phase 3 |
|
42 |
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease |
Completed |
NCT00376168
|
Phase 3 |
Plant cell expressed recombinant glucocerebrosidase (prGCD);Plant cell expressed recombinant glucocerebrosidase (prGCD) |
43 |
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial |
Completed |
NCT00705939
|
Phase 3 |
Taliglucerase alfa |
44 |
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease |
Completed |
NCT00553631
|
Phase 3 |
|
45 |
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase |
Completed |
NCT00478647
|
Phase 2, Phase 3 |
|
46 |
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease |
Completed |
NCT00430625
|
Phase 3 |
|
47 |
Open-Label Phase 3 Long-Term Safety Study of Migalastat |
Completed |
NCT01458119
|
Phase 3 |
migalastat HCl 150mg |
48 |
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase |
Completed |
NCT00712348
|
Phase 3 |
Taliglucerase alfa |
49 |
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment |
Completed |
NCT02055118
|
Phase 2, Phase 3 |
|
50 |
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) |
Completed |
NCT02230566
|
Phase 3 |
UX003 |
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:
|