Details of the Drug-Related molecule(s) Expression Atlas

General Information of Drug (ID: DMWL2XD)

Drug Name

GSK2402968 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
Duchenne dystrophy	8C70	Phase 3	[1]

Cross-matching ID

Molecule(s) Related to This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Dystrophin messenger RNA (DMD mRNA)	TTWLFXU	DMD_HUMAN	Modulator	[2]

------------------------------------------------------------------------------------

The Expression Level of Molecule(s) in Normal Tissue of Major ADME-Related Organs

Molecule	Molecule Type	Gene Name	Liver	Colon	Kidney	Small Intestine
Dystrophin messenger RNA (DMD mRNA)	DTT	DMD	6.356	5.446	5.459	5.326

Molecule Expression Atlas in Normal Tissue of Major ADME-related organs

The Expression Level of Molecule(s) between Disease Section and Healthy Individual Tissue

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 between Disease Section and Healthy Individual Tissue

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.