Drug Information

Drug General Information
Drug ID
D0KX8B
Former ID
DIB009618
Drug Name
GNE Lipoplex
Synonyms
GNE Lipoplex (intramuscular, HIBM-2)
Indication Myopathy [ICD10:M60-M63] Phase 1 [549283]
Company
Gradalis
Target and Pathway
Target(s) N-acetylmannosamine kinase Target Info Inhibitor [531448]
BioCyc Pathway CMP-N-acetylneuraminate biosynthesis I (eukaryotes)
KEGG Pathway Amino sugar and nucleotide sugar metabolism
Metabolic pathways
PathWhiz Pathway Amino Sugar Metabolism
Reactome Sialic acid metabolism
References
Ref 549283Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800034673)
Ref 531448Hereditary inclusion body myopathy: single patient response to intravenous dosing of GNE gene lipoplex. Hum Gene Ther. 2011 Nov;22(11):1331-41.

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