MCID: ADL071
MIFTS: 15

Adult Krabbe Disease

Categories: Neuronal diseases, Eye diseases, Metabolic diseases, Rare diseases, Genetic diseases

Aliases & Classifications for Adult Krabbe Disease

MalaCards integrated aliases for Adult Krabbe Disease:

Name: Adult Krabbe Disease 56

Classifications:



External Ids:

Orphanet 56 ORPHA206448
UMLS via Orphanet 70 C0268252
ICD10 via Orphanet 34 E75.2

Summaries for Adult Krabbe Disease

MalaCards based summary : Adult Krabbe Disease is related to krabbe disease. An important gene associated with Adult Krabbe Disease is GALC (Galactosylceramidase). The drugs Cyclophosphamide and Busulfan have been mentioned in the context of this disorder. Affiliated tissues include bone, eye and bone marrow.

Related Diseases for Adult Krabbe Disease

Diseases in the Krabbe Disease family:

Infantile Krabbe Disease Late-Infantile/juvenile Krabbe Disease
Adult Krabbe Disease

Diseases related to Adult Krabbe Disease via text searches within MalaCards or GeneCards Suite gene sharing:

id Related Disease Score Top Affiliating Genes
1 krabbe disease 9.7

Symptoms & Phenotypes for Adult Krabbe Disease

Drugs & Therapeutics for Adult Krabbe Disease

Drugs for Adult Krabbe Disease (from DrugBank, HMDB, Dgidb, PharmGKB, IUPHAR, NovoSeek, BitterDB):

(show all 47)
id Name Status Phase Clinical Trials Cas Number PubChem Id
1
Cyclophosphamide Approved, Investigational Phase 2, Phase 3 50-18-0, 6055-19-2 2907
2
Busulfan Approved, Investigational Phase 2, Phase 3 55-98-1 2478
3
Prednisolone Approved, Vet_approved Phase 2, Phase 3 50-24-8 5755
4
Methylprednisolone Approved, Vet_approved Phase 2, Phase 3 83-43-2 6741
5 Prednisolone acetate Phase 2, Phase 3
6 Alkylating Agents Phase 2, Phase 3
7 Methylprednisolone acetate Phase 2, Phase 3
8 Methylprednisolone Hemisuccinate Phase 2, Phase 3
9 Immunosuppressive Agents Phase 2, Phase 3
10 Prednisolone hemisuccinate Phase 2, Phase 3
11 Prednisolone phosphate Phase 2, Phase 3
12 Antilymphocyte Serum Phase 2, Phase 3
13 Antirheumatic Agents Phase 2, Phase 3
14
Melphalan Approved Phase 2 148-82-3 4053 460612
15
Mycophenolic acid Approved Phase 2 24280-93-1 446541
16
Mycophenolate mofetil Approved, Investigational Phase 2 128794-94-5 5281078
17
Hydroxyurea Approved Phase 2 127-07-1 3657
18
Miconazole Approved, Investigational, Vet_approved Phase 2 22916-47-8 4189
19
alemtuzumab Approved, Investigational Phase 2 216503-57-0
20
Clofarabine Approved, Investigational Phase 2 123318-82-1 119182
21
Benzocaine Approved Phase 2 1994-09-7, 94-09-7 2337
22
Celecoxib Approved, Investigational Phase 2 169590-42-5 2662
23
Fludarabine Approved Phase 2 21679-14-1, 75607-67-9 30751
24
rituximab Approved Phase 2 174722-31-7 10201696
25 Thiotepa Approved Phase 2 52-24-4 5453
26
Acetylcysteine Approved, Investigational Phase 2 616-91-1 12035
27
Mesna Approved Phase 2 3375-50-6 598
28 tannic acid Approved, Nutraceutical Phase 2
29 Tocopherol Approved, Nutraceutical Phase 2
30
Vitamin E Approved, Nutraceutical, Vet_approved Phase 2 59-02-9 14985
31 Cyclosporins Phase 2
32 Dermatologic Agents Phase 2
33 Nucleic Acid Synthesis Inhibitors Phase 2
34 Anti-Bacterial Agents Phase 2
35 Anti-Infective Agents Phase 2
36 Antifungal Agents Phase 2
37 Antibiotics, Antitubercular Phase 2
38 Antimetabolites Phase 2
39 Antimetabolites, Antineoplastic Phase 2
40 Calcineurin Inhibitors Phase 2
41 N-monoacetylcystine Phase 2
42 Thioctic Acid Phase 2
43 Tocopherols Phase 2
44 Tocotrienols Phase 2
45 Vitamins Phase 2
46 Tocotrienol Investigational, Nutraceutical Phase 2 6829-55-6
47 Alpha-lipoic Acid Nutraceutical Phase 2

Interventional clinical trials:

(show all 18)

id Name Status NCT ID Phase Drugs
1 Stem Cell Transplant for Inborn Errors of Metabolism Completed NCT00176904 Phase 2, Phase 3 Busulfan, Cyclophosphamide, Antithymocyte Globulin
2 HSCT for High Risk Inherited Inborn Errors Completed NCT00383448 Phase 2 Clofarabine;Melphalan;Alemtuzumab;mycophenylate mofetil;Hydroxyurea
3 Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Recruiting NCT01962415 Phase 2 Hydroxyurea;Alemtuzumab;Fludarabine;Melphalan;Thiotepa
4 MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Recruiting NCT02171104 Phase 2 IMD Preparative Regimen;Osteopetrosis Only Preparative Regimen;Osteopetrosis Haploidentical Only Preparative Regimen;cALD SR-A (Standard-Risk, Regimen A);cALD SR-B (Standard-Risk, Regimen B);cALD HR-D (High-Risk, Regimen C);cALD HR-D (High-Risk, Regimen D)
5 Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Active, not recruiting NCT01043640 Phase 2 Campath-1H;Cyclophosphamide;Busulfan;Cyclosporine A;Mycophenolate Mofetil
6 Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders Active, not recruiting NCT01372228 Phase 1, Phase 2
7 Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Terminated NCT00668564 Phase 2 Cyclophosphamide;Campath-1H;Busulfan
8 Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Withdrawn NCT02715505 Phase 1, Phase 2 Umbilical cord blood transplantation with HSC835
9 Human Placental-Derived Stem Cell Transplantation Recruiting NCT01586455 Phase 1 Human Placental Derived Stem Cell
10 UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Recruiting NCT02254863 Phase 1
11 Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children Unknown status NCT01938014
12 Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation Unknown status NCT00005900
13 Diagnostic and Screening Study of Genetic Disorders Completed NCT00006057
14 Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Completed NCT01626092 Campath-1H;Clofarabine;Melphalan;Cyclosporine A;Mycophenolate mofetil
15 Quality Assessment Creatinines in Plasma and Urine Completed NCT01575392
16 Biomarker for Krabbe Disease Recruiting NCT01425489
17 Krabbe Disease Global Patient Registry Recruiting NCT02993796
18 Genetic Characterization of Movement Disorders and Dementias Recruiting NCT02014246

Search NIH Clinical Center for Adult Krabbe Disease

Genetic Tests for Adult Krabbe Disease

Anatomical Context for Adult Krabbe Disease

MalaCards organs/tissues related to Adult Krabbe Disease:

39
Bone, Eye, Bone Marrow

Publications for Adult Krabbe Disease

Articles related to Adult Krabbe Disease:

id Title Authors Year
1
MR imaging and proton MR spectroscopy in adult Krabbe disease. ( 11003282 )
2000

Variations for Adult Krabbe Disease

Expression for Adult Krabbe Disease

Search GEO for disease gene expression data for Adult Krabbe Disease.

Pathways for Adult Krabbe Disease

GO Terms for Adult Krabbe Disease

Sources for Adult Krabbe Disease

3 CDC
7 CNVD
9 Cosmic
10 dbSNP
11 DGIdb
16 ExPASy
18 FMA
28 GO
29 GTR
30 HGMD
31 HMDB
32 HPO
33 ICD10
34 ICD10 via Orphanet
35 ICD9CM
36 IUPHAR
37 KEGG
38 LifeMap
40 MedGen
42 MeSH
43 MESH via Orphanet
44 MGI
46 NCI
47 NCIt
48 NDF-RT
51 NINDS
52 Novoseek
54 OMIM
55 OMIM via Orphanet
59 PubMed
60 QIAGEN
65 SNOMED-CT via HPO
66 SNOMED-CT via Orphanet
67 TGDB
68 Tocris
69 UMLS
70 UMLS via Orphanet
Content
Loading form....