Details of the Drug

General Information of Drug (ID: DMWL2XD)

Drug Name

GSK2402968

Indication

Disease Entry	ICD 11	Status	REF
Duchenne dystrophy	8C70	Phase 3	[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
Dystrophin messenger RNA (DMD mRNA)	TTWLFXU	DMD_HUMAN	Modulator	[2]

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Molecular Interaction Atlas (MIA)

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Molecular Expression Atlas of This Drug

The Studied Disease

Duchenne dystrophy

ICD Disease Classification

8C70

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Dystrophin messenger RNA (DMD mRNA)	DTT	DMD	1.15E-07	-1.07	-2.94

Molecular Expression Atlas (MEA)

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References

1	ClinicalTrials.gov (NCT01480245) Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy. U.S. National Institutes of Health.
2	Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014 Oct;13(10):987-96.