Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DK3U6C
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| Drug Name |
BMN 250
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| Synonyms |
tralesinidase alfa
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| Drug Type |
Fusion protein
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| Indication | Mucopolysaccharidosis type IIIB [ICD-11: 5C56.3Y] | Phase 1/2 | [1] | |
| Company |
BioMarin Pharmaceutical
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Alpha-N-acetylglucosaminidase (NAGLU) | Target Info | Replacement | [2] |
| KEGG Pathway | Glycosaminoglycan degradation | |||
| Metabolic pathways | ||||
| Lysosome | ||||
| Reactome | HS-GAG degradation | |||
| WikiPathways | Glycosaminoglycan metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B. Drug Deliv Transl Res. 2020 Apr;10(2):425-439. | |||
| REF 2 | Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B. Drug Deliv Transl Res. 2020 Apr;10(2):425-439. | |||
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