Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0OQ9V
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| Former ID |
DIB001925
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| Drug Name |
BMN-110
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| Synonyms |
Enzyme replacement therapy (iv infusion, MPS IVA), BioMarin; Enzyme replacement therapy (iv infusion, Morquio A syndrome), BioMarin; Enzyme replacement therapy (iv infusion, mucopolysaccharidosis IVA), BioMarin; N-acetylgalactosamine-6-sulfatase (GALNS) (Morquio A syndrome), BioMarin
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| Indication | Morquio syndrome [ICD-11: 5C56.32; ICD-10: E76.2] | Phase 3 | [1] | |
| Company |
Biomarin pharmaceutical
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | N-acetylgalactosamine 6 sulfatase (GALNS) | Target Info | Modulator | [2] |
| BioCyc | Chondroitin sulfate degradation (metazoa) | |||
| KEGG Pathway | Glycosaminoglycan degradation | |||
| Metabolic pathways | ||||
| Lysosome | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT01415427) Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome). U.S. National Institutes of Health. | |||
| REF 2 | Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014 Nov;37(6):979-90. | |||
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