Details of the Drug

General Information of Drug (ID: DM94Y6O)

Drug Name

BMN-701

Synonyms

IGF2-GAA; XC-701; ZC-701; Enzyme replacement therapy (GILT, Pompe disease), BioMarin/ZyStor

Indication

Disease Entry	ICD 11	Status	REF
Pompe disease	5C51.3	Phase 3	[1]
Type 2 glycogen storage disease	5C51.3	Phase 3	[2]
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Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Lysosomal alpha-glucosidase (GAA)	TTLPC70	LYAG_HUMAN	Modulator	[3]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT01924845) BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study). U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
3	Clinical pipeline report, company report or official report of BioMarin Pharma.