Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DQ62VF
|
|||
| Drug Name |
SOBI003
|
|||
| Drug Type |
Recombinant protein
|
|||
| Indication | Sanfilippo syndrome [ICD-11: 5C56.3Y] | Phase 1/2 | [1] | |
| Company |
Swedish Orphan Biovitrum
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | N-sulphoglucosamine sulphohydrolase (SGSH) | Target Info | Replacement | [2] |
| KEGG Pathway | Glycosaminoglycan degradation | |||
| Metabolic pathways | ||||
| Lysosome | ||||
| Reactome | HS-GAG degradation | |||
| WikiPathways | Glycosaminoglycan metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03811028) A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients. U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Swedish Orphan Biovitrum. | |||
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