Details of the Drug

General Information of Drug (ID: DMXNTR7)

Drug Name

SOBI003

Indication

Disease Entry	ICD 11	Status	REF
Sanfilippo syndrome	5C56.3Y	Phase 1/2	[1]
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Drug Type

Recombinant protein

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
N-sulphoglucosamine sulphohydrolase (SGSH)	TTPJ2SH	SPHM_HUMAN	Replacement	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

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.
2	Clinical pipeline report, company report or official report of Swedish Orphan Biovitrum.