General Information of Drug (ID: DMHZ4JI)

Drug Name
HGT-1410 Drug Info
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 Drug ID
DMHZ4JI

Molecule-Related Drug Atlas

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Clinical Trial Drug(s)
Drug Name Drug ID Indication ICD 11 Highest Status REF
LYS-SAF302 DMUQWRD Mucopolysaccharidosis 5C56.3 Phase 2/3 [3]
ABO-102 DMFJB35 Mucopolysaccharidosis 5C56.3 Phase 1/2 [4]
SOBI003 DMXNTR7 Sanfilippo syndrome 5C56.3Y Phase 1/2 [5]

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
N-sulphoglucosamine sulphohydrolase (SGSH) TTPJ2SH SPHM_HUMAN Modulator [2]

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.
3 Clinical pipeline report, company report or official report of Lysogene.
4 Clinical pipeline report, company report or official report of Abeona Therapeutics.
5 Clinical pipeline report, company report or official report of Swedish Orphan Biovitrum.