Details of the Drug

General Information of Drug (ID: DMHZ4JI)

Drug Name

HGT-1410

Synonyms

Intrathecal sulfamidase replacement therapy (FGE, Sanfilippo syndrome), Shire; Intrathecal heparin n-sulfamidase (FGE, Sanfilippo syndrome), Shire

Indication

Disease Entry	ICD 11	Status	REF
Sanfilippo syndrome	5C56.3Y	Phase 2	[1]
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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
N-sulphoglucosamine sulphohydrolase (SGSH)	TTPJ2SH	SPHM_HUMAN	Modulator	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

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.
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.