Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0E5RA
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| Former ID |
DIB000850
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| Drug Name |
HGT-1111
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| Synonyms |
Metazym; M-0001; Recombinant arylsulfatase A (lysosomal storage disease), Shire; Recombinant arylsulfatase A (lysosomal storage disease), Zymenex; Recombinant arylsulfatase A (metachromatic leukodystrophy), Shire; Lysosomal arylsulfatase A replacement therapy (FGE, metachromatic leukodystrophy), Shire; Lysosomal asa replacement therapy (FGE, metachromatic leukodystrophy), Shire
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| Indication | Metachromatic leukodystrophy [ICD-11: 5C56.02] | Phase 2 | [1] | |
| Company |
Zymenex A/S
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Cerebroside-sulfatase (ARSA) | Target Info | Modulator | [1] |
| KEGG Pathway | Sphingolipid metabolism | |||
| Lysosome | ||||
| Pathwhiz Pathway | Sphingolipid Metabolism | |||
| Reactome | Glycosphingolipid metabolism | |||
| WikiPathways | Sphingolipid metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT00681811) Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD. U.S. National Institutes of Health. | |||
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