Details of the Drug

General Information of Drug (ID: DMURO94)

Drug Name
GNE Lipoplex
Synonyms GNE Lipoplex (intramuscular, HIBM-2)
Indication
Disease Entry ICD 11 Status REF
Myopathy 8C7Y Phase 1 [1]
Cross-matching ID
TTD ID
D0KX8B

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
N-acetylmannosamine kinase (GNE) TT4DP5S GLCNE_HUMAN Inhibitor [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

Molecular Expression Atlas of This Drug

ICD Disease Classification 08 Disease of the nervous system
Disease Class ICD-11: 8A00 Parkinsonism
The Studied Tissue Muscle tissue
The Studied Disease Myopathy [ICD-11:8A00.0]
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
N-acetylmannosamine kinase (GNE) DTT GNE 7.10E-01 -0.02 -0.04
Molecular Expression Atlas (MEA) Jump to Detail Molecular Expression Atlas of This Drug

References

1 Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800034673)
2 Hereditary inclusion body myopathy: single patient response to intravenous dosing of GNE gene lipoplex. Hum Gene Ther. 2011 Nov;22(11):1331-41.