Drugs for Lysosomal Storage Disease (from DrugBank, HMDB, Dgidb, PharmGKB, IUPHAR, NovoSeek, BitterDB):
(show all 31)
# |
|
Name |
Status |
Phase |
Clinical Trials |
Cas Number |
PubChem Id |
1 |
|
Busulfan |
Approved, Investigational |
Phase 2 |
|
55-98-1 |
2478 |
Synonyms:
1,4-Bis(methanesulfonoxy)butane
1,4-Bis(methanesulphonoxy)butane
1,4-Butanediol dimethanesulfonate
1,4-Butanediol dimethanesulfonic acid
1,4-Butanediol dimethanesulphonate
1,4-Butanediol dimethanesulphonic acid
1,4-Dimesyloxybutane
1,4-Dimethanesulfonoxybutane
1,4-Dimethanesulphonoxybutane
Bisulfex
Bisulphex
BUSILVEX
BUSULFAN
Busulfan glaxosmithkline brand
Busulfan orphan brand
Busulfan wellcome
Busulfan wellcome brand
Busulfano
Busulfanum
Busulfex
BUSULFEX IV
Busulphan
Busulphane
Busulphano
Busulphanum
Butanedioldimethanesulfonate
Buzulfan
Citosulfan
Glaxo wellcome brand OF busulfan
GlaxoSmithKline brand OF busulfan
Glyzophrol
Leucosulfan
|
Leucosulphan
Mablin
Mielevcin
Mielosan
Mielucin
Milecitan
Mileran
Misulban
Mitosan
Mitostan
Myeleukon
Myeloleukon
Myelosan
Mylecytan
Myleran
Myléran
Myleran Tablets
N-Butane-1,3-di(methylsulfonate)
NCI-C01592
NSC-750
Orphan brand OF busulfan
Sulfabutin
Sulphabutin
Tetramethylene bis(methanesulfonate)
Tetramethylene bis(methanesulfonic acid)
Tetramethylene bis(methanesulphonate)
Tetramethylene bis(methanesulphonic acid)
Tetramethylene dimethane sulfonate
Tetramethylenester kyseliny methansulfonove
Wellcome brand OF busulfan
Wellcome, busulfan
|
|
2 |
|
Melphalan |
Approved |
Phase 1 |
|
148-82-3 |
4053 460612 |
Synonyms:
2-Amino-3-[4-[bis(2-chloroethyl)amino]phenyl]propanoate
2-Amino-3-{4-[bis(2-chloroethyl)amino]phenyl}propanoate
3-(p-(Bis(2-chloroethyl)amino)phenyl)-L-alanine
3-p-(Di(2-chloroethyl)amino)-phenyl-L-alanine
4-(Bis(2-chloroethyl)amino)-L-phenylalanine
4-(Bis(2-chloroethyl)amino)phenylalanine
AIKERAN
ALANINE NITROGEN MUSTARD
Alkeran
CB-3025
L-3-(p-(Bis(2-chloroethyl)amino)phenyl)alanine
Levofalan
L-PAM
L-Phenylalanine mustard
L-PHENYLALANINE MUSTARD (L-PAM)
L-SARCOLYSIN
L-Sarcolysine
L-Sarkolysin
Medphalan
MELFALAN
Melfalano
Melphalan
|
Melphalanum
Mephalan
Merphalan
MLP673
Mustard, phenylalanine
NCI-C04853
NSC-241286
NSC-8806
p-Bis(b-chloroethyl)aminophenylalanine
p-Bis(beta-chloroethyl)aminophenylalanine
p-Bis(β-chloroethyl)aminophenylalanine
p-Di-(2-chloroethyl)amino-L-phenylalanine
Phenylalanine mustard
Phenylalanine nitrogen mustard
p-L-Sarcolysin
p-N,N-Bis(2-chloroethyl)amino-L-phenylalanine
p-N-Bis(2-chloroethyl)amino-L-phenylalanine
Sarcolysin
Sarcolysine
Sarkolysin
SK 15673
SK-15673
|
|
3 |
|
Fludarabine |
Approved |
Phase 1 |
|
75607-67-9, 21679-14-1 |
30751 657237 |
Synonyms:
2-F-ARAA
2-F-Ara-A|CCRIS 3382|F-Ara-A|Fludara®
2F-Ara-AMP
2-fluoro ARA-A
2-Fluoroadenine arabinoside 5'-monophosphate
2-Fluoroadenine arabinoside 5'-monophosphoric acid
2-Fluoro-ara AMP
2-FLUORO-ARA-A
9 beta-D-Arabinofuranosyl-2-fluoroadenine monophosphate
9-b-Arabinofuranosyl-2-fluoroadenine-5'-phosphate
9-b-Arabinofuranosyl-2-fluoroadenine-5'-phosphoric acid
9-b-D-Arabinofuranosyl-2-fluoroadenine 5'-(dihydrogen phosphate)
9-b-D-Arabinofuranosyl-2-fluoroadenine 5'-(dihydrogen phosphoric acid)
9-b-D-Arabinofuranosyl-2-fluoroadenine 5'-monophosphate
9-b-D-Arabinofuranosyl-2-fluoroadenine 5'-monophosphoric acid
9-beta-Arabinofuranosyl-2-fluoroadenine-5'-phosphate
9-beta-Arabinofuranosyl-2-fluoroadenine-5'-phosphoric acid
9-beta-D-Arabinofuranosyl-2-fluoroadenine 5'-(dihydrogen phosphate)
9-beta-D-Arabinofuranosyl-2-fluoroadenine 5'-(dihydrogen phosphoric acid)
9-beta-D-Arabinofuranosyl-2-fluoroadenine 5'-monophosphate
9-beta-D-Arabinofuranosyl-2-fluoroadenine 5'-monophosphoric acid
9-Β-arabinofuranosyl-2-fluoroadenine-5'-phosphate
9-Β-arabinofuranosyl-2-fluoroadenine-5'-phosphoric acid
|
9-Β-D-arabinofuranosyl-2-fluoroadenine 5'-(dihydrogen phosphate)
9-Β-D-arabinofuranosyl-2-fluoroadenine 5'-(dihydrogen phosphoric acid)
9-Β-D-arabinofuranosyl-2-fluoroadenine 5'-monophosphate
9-Β-D-arabinofuranosyl-2-fluoroadenine 5'-monophosphoric acid
Beneflur
FAMP
FaraAMP
F-Ara-AMP
Fludara
Fludarabina
FLUDARABINE
Fludarabine 5'-monophosphate
Fludarabine 5'-monophosphoric acid
Fludarabine monophosphate
Fludarabine monophosphoric acid
Fludarabine phosphate
Fludarabine phosphoric acid
Fludarabinum
Fludura
Fluoro-ara-AMP
NSC-312887
NSC-328002
OFORTA
|
|
4 |
|
Alemtuzumab |
Approved, Investigational |
Phase 1 |
|
216503-57-0 |
|
Synonyms:
ALEMTUZUMAB
ALEMTUZUMAB (GENETICAL RECOMBINATION)
CAMPATH
campath 1H|Campath®|campath-1H|Lemtrada®
CAMPATH MABCAMPATH
CAMPATH MABCAMPATH, LEMTRADA
|
CAMPATH-1H
GZ402673
GZ-402673
LDP-03
LEMTRADA
MABCAMPATH
|
|
5 |
|
Thiotepa |
Approved, Investigational |
Phase 1 |
|
52-24-4 |
5453 |
Synonyms:
Girostan
NSC-6396
Rethio
Tepadina
Tepadina®|Thioplex®
Tespa
Tespamin
Thio tepa
|
Thiophosphamide
Thioplex
Thio-tepa
THIOTEPA
THIOTEPA 或 THIOTEPA
Triethylenethiophosphoramide
Tris(1-aziridinyl)phosphine sulfide
|
|
6 |
|
Hydroxyurea |
Approved |
Phase 1 |
|
127-07-1 |
3657 |
Synonyms:
Biosupressin
Carbamohydroxamate
Carbamohydroxamic acid
Carbamohydroximate
Carbamohydroximic acid
Carbamohydroxyamic acid
Carbamoyl oxime
Carbamyl hydroxamate
Carbamyl hydroxamic acid
Droxia
Droxia®|Hydrea®|hydroxycarbamide|Siklos®|SQ-1089|Xromi®
Hidrix
Hidroxicarbamida
HU
Hydrea
Hydreia
Hydroxicarbamidum
Hydroxycarbamid
Hydroxycarbamide
Hydroxycarbamidum
|
Hydroxycarbamine
Hydroxyharnstoff
Hydroxylurea
HYDROXYUREA
Hydura
Hydurea
Idrossicarbamide
Idrossicarbamide [Dcit]
Litaler
Litalir
N-Carbamoylhydroxylamine
N-HYDROXYUREA
NSC-32065
Oncocarbide
Onco-Carbide
Oxyurea
SIKLOS
SQ-1089
Sterile Urea
Ureaphil
|
|
7 |
|
Alkylating Agents |
|
Phase 1 |
|
|
|
8 |
|
Antineoplastic Agents, Alkylating |
|
Phase 1 |
|
|
|
9 |
|
Immunosuppressive Agents |
|
Phase 1 |
|
|
|
10 |
|
Pharmaceutical Solutions |
|
Phase 1 |
|
|
|
11 |
|
Benzocaine |
Approved, Investigational |
|
|
1994-09-7, 94-09-7 |
2337 |
Synonyms:
(P-(Ethoxycarbonyl)phenylamine
4-(Ethoxycarbonyl)aniline
4-(Ethoxycarbonyl)phenylamine
4-Aminobenzoate
4-Aminobenzoate ethyl ester
4-Aminobenzoic acid
4-Aminobenzoic acid ethyl ester
4-Carbethoxyaniline
Acetate, benzocaine
Aethoform
AEZODENT
Amben ethyl ester
Americaine
Anaesthan-syngala
Anaesthesin
Anaesthin
Anestezin
Anesthesin
Anesthesine
Anesthone
ANTHISAN PLUS
AR01
AR-01
Baby anbesol
Bensokain
Benzoak
Benzocaina
BENZOCAINE
Benzocaine acetate
Benzocaine formate
Benzocaine hydrobromide
Benzocaine hydrochloride
Benzocaine methanesulfonate
Benzocainum
BURNEZE
CHILDREN'S CHLORASEPTIC
DEQUACAINE
Dermoplast
Diet ayds
Ethoform
Ethyl 4-aminobenzoate
|
Ethyl 4-aminobenzoic acid
Ethyl aminobenzoate
Ethyl aminobenzoic acid
Ethyl P-aminobenzenecarboxylate
Ethyl p-aminobenzoate
Ethyl p-aminobenzoic acid
Ethyl p-aminophenylcarboxylate
Ethyl p-aminophenylcarboxylic acid
Ethylester kyseliny P-aminobenzoove
Formate, benzocaine
Hurricaine
Hydrobromide, benzocaine
Hydrochloride, benzocaine
Identhesin
Keloform
LANACANE
MEDILAVE
Methanesulfonate, benzocaine
NESTOSYL
Norcain
Norcaine
NSC-41531
NSC-4688
Orabase-b
Ora-jel
ORAJEL
Orthesin
p-(Ethoxycarbonyl)aniline
P-Aminobenzoate
P-Aminobenzoic acid
P-Aminobenzoic acid ethyl ester
Parathesin
Parathesine
p-Carbethoxyaniline
p-Ethoxycarboxylic aniline
Slim mint gum
Solarcaine
Solu H
Topcaine
TYROSOLVEN
TYROZETS
|
|
12 |
|
Tannic acid |
Approved |
|
|
1401-55-4 |
16129878 16129778 |
Synonyms:
Acid, tannic
ácido tánico
Acids, tannic
E181
FEMA NO. 3042
Gallotannic acid
|
Gallotannin
NSC-656273
TANNIC ACID
Tannic acids
Tannins
|
|
13 |
|
Mycophenolic acid |
Approved, Investigational |
|
|
24280-93-1 |
446541 |
Synonyms:
(e)-6-(4-Hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoate
(e)-6-(4-Hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoic acid
68618
Acid, mycophenolic
Acide mycophenolique
Acido micofenolico
Acidum mycophenolicum
Cellcept
ERL080
ERL-080
ERL-080|Myfortic® (mycophenolate sodium)
Melbex
Micofenolico acido
Mofetil hydrochloride, mycophenolate
Mofetil, mycophenolate
|
Mycophenoic acid
Mycophenolate
Mycophenolate mofetil
Mycophenolate mofetil hydrochloride
Mycophenolate sodium
Mycophenolate, sodium
MYCOPHENOLIC ACID
Mycophenolic acid morpholinoethyl ester
Mycophenolsaeure
Mycophenolsäure
Myfortic
NSC-129185
RS-61443 [AS MOFETIL]
Sodium mycophenolate
|
|
14 |
|
Miconazole |
Approved, Investigational, Vet_approved |
|
|
22916-47-8 |
4189 |
Synonyms:
1-(2,4-Dichloro-beta-((2,4-dichlorobenzyl)oxy)phenethyl)imidazole
1-[2-(2,4-Dichloro-benzyloxy)-2-(2,4-dichloro-phenyl)-ethyl]-1H-imidazole
Brentan
CONOFITE
Dactarin
DERMAZOLE
DUMICOAT
Esteve brand OF miconazole
florid(nitrate)|Monistat®|R-14889
Janssen cilag brand OF miconazole
Janssen-cilag brand OF miconazole
Johnston and johnston brand OF miconazole nitrate
LORAMYC
MCZ
MICATIN
|
Miconasil nitrate
Miconazol
Miconazole
Miconazole esteve brand
Miconazole janssen-cilag brand
Miconazole nitrate
Miconazolum
Monistat
MONISTAT-3
MONISTAT-DERM
Nitrate, miconasil
Nitrate, miconazole
NSC-170986
Oravig
VUSION
|
|
15 |
|
Clotrimazole |
Approved, Vet_approved |
|
|
23593-75-1 |
2812 |
Synonyms:
(Chlorotrityl)imidazole
1-((2-Chlorophenyl)diphenylmethyl)-1H-imidazole
1-(a-(2-Chlorophenyl)benzhydryl)imidazole
1-(alpha-(2-Chlorophenyl)benzhydryl)imidazole
1-(O-Chloro-a,a-diphenylbenzyl)imidazole
1-(O-Chloro-alpha,alpha-diphenylbenzyl)imidazole
1-(O-Chlorotrityl)imidazole
1-(O-Chloro-α,α-diphenylbenzyl)imidazole
1-(Α-(2-chlorophenyl)benzhydryl)imidazole
ABTRIM
ACTAVALL
Bay b 5097
BAY-5097
Bayer brand 1 OF clotrimazole
Bayer brand 2 OF clotrimazole
CANDIDEN
Canesten
Canesten 1-day cream combi-pak
Canesten 1-day therapy
Canesten 3-day therapy
Canesten 6-day therapy
CANESTEN AF
Canesten combi-pak 1-day therapy
Canesten combi-pak 3-day therapy
Canesten cream
CANESTEN CRM COMBI
CANESTEN INTERNAL
Canesten solution
CANESTEN VC
Canesten®|Lotrimin®
Canestine
Chlotrimazole
Clotrimaderm
Clotrimaderm cream
Clotrimazol
CLOTRIMAZOLE
Clotrimazole schering brand
Clotrimazolum
Desamix F
Empecid
FB b 5097
Fem care
FemCare
FUNGEDERM
GNF-PF-3499
Gyne lotrimin
Gyne-lotrimin
Gyne-lotrimin 3
Gyne-lotrimin 3 combination pack
Gyne-lotrimin combination pack
Gyne-lotrimin3
Gyne-lotrimin3 combination pack
|
Gynix
Kanesten
Klotrimazole
Lopac-C-6019
Lotrimax
Lotrimin
LOTRIMIN (TN)
Lotrimin af
Lotrimin af cream
Lotrimin af jock-itch cream
Lotrimin af lotion
Lotrimin af solution
Lotrimin cream
Lotrimin lotion
Lotrimin solution
Lotrisone
MASNODERM
mono-Baycuten
Monobaycuten
Mycelax
Mycelex
MYCELEX (TN)
Mycelex 7
Mycelex cream
Mycelex g
Mycelex otc
Mycelex solution
Mycelex troches
Mycelex twin pack
Mycelex: mycosporinrimazole
Mycelex-7
Mycelex-7 combination pack
Mycelex-g
MYCIL GOLD
Myclo cream
Myclo solution
Myclo spray solution
Myclo-gyne
Mycosporin
Mykosporin
Neo-zol cream
NSC-257473
Otomax
Pedisafe
Prestwick_120
PRIVACOM
Rimazole
Schering brand OF clotrimazole
Tibatin
Trimysten
Trivagizole 3
Veltrim
|
|
16 |
|
Clofarabine |
Approved, Investigational |
|
|
123318-82-1 |
119182 |
Synonyms:
(2R,3R,4S,5R)-5-(6-Amino-2-chloropurin-9-yl)-4-fluoro-2-(hydroxymethyl)oxolan-3-ol
2 Chloro 2' arabino fluoro 2' deoxyadenosine
2 Chloro 2' fluoroarabino 2' deoxyadenosine
2-Chloro-2'-arabino-fluoro-2'-deoxyadenosine
2-Chloro-2'-fluoroarabino-2'-deoxyadenosine
2-Chloro-9-(2-deoxy-2-fluoroarabinofuranosyl)adenine
2-Chloro-9-(2'-deoxy-2'-fluoro-b-D-arabinofuranosyl)adenine
2-Chloro-9-(2-deoxy-2-fluoro-b-D-arabinofuranosyl)adenine
2-Chloro-9-(2'-deoxy-2'-fluoro-beta-D-arabinofuranosyl)adenine
2-Chloro-9-(2-deoxy-2-fluoro-beta-D-arabinofuranosyl)adenine
2-Chloro-9-(2-deoxy-2-fluoro-beta-D-arbinofuranosyl)adenine
2-Chloro-9-(2'-deoxy-2'-fluoro-β-D-arabinofuranosyl)adenine
|
2-Chloro-9-(2-deoxy-2-fluoro-β-D-arabinofuranosyl)adenine
CAFdA
CAFdA|Cl-F-araA|Clolar®|Evoltra®
CL-F-Ara-a
CL-F-ARAA
CLOFARABIN
Clofarabina
CLOFARABINE
Clofarabinum
Clofarex
Clolar
Evoltra
|
|
17 |
|
Antirheumatic Agents |
|
|
|
|
|
18 |
|
Anti-Bacterial Agents |
|
|
|
|
|
19 |
|
Anti-Infective Agents |
|
|
|
|
|
20 |
|
Calcineurin Inhibitors |
|
|
|
|
|
21 |
|
Cyclosporins |
|
|
|
|
|
22 |
|
Antifungal Agents |
|
|
|
|
|
23 |
|
Antimetabolites |
|
|
|
|
|
24 |
|
Antitubercular Agents |
|
|
|
|
|
25 |
|
Antibiotics, Antitubercular |
|
|
|
|
|
26 |
|
Antineoplastic Agents, Immunological |
|
|
|
|
|
27 |
|
Dermatologic Agents |
|
|
|
|
|
28 |
|
Liver Extracts |
|
|
|
|
|
29 |
|
Immunoglobulins |
|
|
|
|
|
30 |
|
Antibodies, Blocking |
|
|
|
|
|
31 |
|
Antibodies |
|
|
|
|
|
Interventional clinical trials:
(show all 36)
# |
Name |
Status |
NCT ID |
Phase |
Drugs |
1 |
An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD). |
Recruiting |
NCT04283227 |
Phase 3 |
|
2 |
A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism |
Terminated |
NCT00654433 |
Phase 3 |
|
3 |
A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector |
Recruiting |
NCT03897361 |
Phase 1, Phase 2 |
|
4 |
A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease |
Recruiting |
NCT04040049 |
Phase 1, Phase 2 |
|
5 |
A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD) |
Active, not recruiting |
NCT03392987 |
Phase 2 |
|
6 |
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy |
Active, not recruiting |
NCT01560182 |
Phase 1, Phase 2 |
|
7 |
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease |
Active, not recruiting |
NCT04093349 |
Phase 1, Phase 2 |
|
8 |
A Pilot Study of Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation |
Completed |
NCT00744692 |
Phase 1 |
Reduced Intensity Conditioning |
9 |
An Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I |
Terminated |
NCT00786968 |
Phase 1 |
laronidase |
10 |
A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I |
Terminated |
NCT00215527 |
Phase 1 |
laronidase |
11 |
Treatment of Early Infantile-Onset Lysosomal Storage Diseases With Fetal Umbilical Cord Blood (UCB) Transplantation |
Withdrawn |
NCT01003912 |
Phase 1 |
|
12 |
Enzymatic and Genotypic Screening of Lysosomal Storage Diseases in Minority Groups |
Unknown status |
NCT03812042 |
|
|
13 |
Investigating Lysosomal Storage Diseases in Minority Groups |
Unknown status |
NCT02120235 |
|
|
14 |
Cellular Pharmacodynamics of Small Molecules in Sanfilippo Disease(s) (MPS3) and Other Lysosomal Storage Disorders |
Unknown status |
NCT03812055 |
|
|
15 |
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children |
Completed |
NCT01938014 |
|
|
16 |
A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I |
Completed |
NCT00852358 |
|
laronidase |
17 |
Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT) |
Completed |
NCT01626092 |
|
Campath-1H;Clofarabine;Melphalan;Cyclosporine A;Mycophenolate mofetil |
18 |
The Nosology and Etiology of Leukodystrophies of Unknown Cause |
Completed |
NCT00001671 |
|
|
19 |
Stimulation of the Human Central and Peripheral Nervous System With a Magnetic Stimulator |
Completed |
NCT00001780 |
|
|
20 |
A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease |
Completed |
NCT03893240 |
|
|
21 |
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease: An International, Multicenter, Epidemiological Protocol |
Completed |
NCT02416661 |
|
|
22 |
A Pilot Study of Diet and Exercise Therapy in Pompe Disease |
Completed |
NCT02363153 |
|
|
23 |
Characterization of the Patient Population With Galactosialidosis |
Completed |
NCT01416467 |
|
|
24 |
Investigations Into Inborn Errors of Cholesterol Synthesis and Related Disorders |
Recruiting |
NCT00046202 |
|
|
25 |
Registry of Patients Diagnosed With Lysosomal Storage Diseases |
Recruiting |
NCT05619900 |
|
|
26 |
Investigation of Molecular and Cellular Mechanisms of Lysosomal Storage Diseases |
Recruiting |
NCT02000310 |
|
|
27 |
A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry |
Recruiting |
NCT04393701 |
|
|
28 |
A Multicentre, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease |
Recruiting |
NCT04455230 |
|
|
29 |
Identification and Characterization of Novel Coding, Splicing and Non-Coding Variants That Contribute to Genetic Disorders |
Recruiting |
NCT04399694 |
|
|
30 |
Biomarker for Maroteaux-Lamy Disease: BioMaroteaux-Lamy AN INTERNATIONAL, MULTICENTER, EPIDEMIOLOGICAL PROTOCOL |
Active, not recruiting |
NCT01458613 |
|
|
31 |
Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score |
Enrolling by invitation |
NCT04943991 |
|
|
32 |
Studies of Genetic Heterogeneity in Patients With Lysosomal Storage Disorders |
Enrolling by invitation |
NCT00001215 |
|
|
33 |
Expanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR αβ+T Cells In Pediatric Patients Affected by Hematological and Other Disorders |
No longer available |
NCT03639844 |
|
rimiducid |
34 |
Natural History Study of Children With Metachromatic Leukodystrophy |
Terminated |
NCT01963650 |
|
|
35 |
A Natural History Study of Aspartylglucosaminuria |
Terminated |
NCT03853876 |
|
|
36 |
Complement Activation in the Lysosomal Storage Disorders |
Withdrawn |
NCT04189601 |
|
|
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:
|