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]
Cross-matching ID
TTD ID
D0Q5BK

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]
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.