Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0Q5BK
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| Former ID |
DIB000525
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| Drug Name |
HGT-1410
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| Synonyms |
Intrathecal sulfamidase replacement therapy (FGE, Sanfilippo syndrome), Shire; Intrathecal heparin n-sulfamidase (FGE, Sanfilippo syndrome), Shire
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| Indication | Sanfilippo syndrome [ICD-11: 5C56.3Y] | Phase 2 | [1] | |
| Company |
Shire Human Genetic Therapies Inc
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | N-sulphoglucosamine sulphohydrolase (SGSH) | Target Info | Modulator | [2] |
| KEGG Pathway | Glycosaminoglycan degradation | |||
| Metabolic pathways | ||||
| Lysosome | ||||
| Reactome | HS-GAG degradation | |||
| WikiPathways | Glycosaminoglycan metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT02350816) Safety and Efficacy Study for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. U.S. National Institutes of Health. | |||
| REF 2 | ClinicalTrials.gov (NCT01155778) Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients. U.S. National Institutes of Health. | |||
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