General Information of Drug (ID: DMFYABR)

Drug Name
PRO-044 Drug Info
Synonyms Exon-44 skipping antisense oligonucleotide (Duchenne muscular dystrophy), Prosensa
Indication
Disease Entry ICD 11 Status REF
Duchenne dystrophy 8C70 Phase 2 [1]
Cross-matching ID
TTD Drug ID
DMFYABR

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 Not Available [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 (NCT02329769) Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD). U.S. National Institutes of Health.
2 Update on neuromuscular disorders in pediatric orthopaedics: Duchenne muscular dystrophy, myelomeningocele, and cerebral palsy. J Pediatr Orthop. 2014 Oct-Nov;34 Suppl 1:S44-8.