Drug Information

Drug General Information
Drug ID
D05DFV
Former ID
DIB004035
Drug Name
PRO-044
Synonyms
Exon-44 skipping antisense oligonucleotide (Duchenne muscular dystrophy), Prosensa
Drug Type
Antisense drug
Indication Duchenne muscular dystrophy [ICD10:G71.0] Phase 2 [525046]
Company
Leiden University Medical Center
Target and Pathway
Target(s) mRNA of Dystrophin Target Info [532949]
KEGG Pathway Hypertrophic cardiomyopathy (HCM)
Arrhythmogenic right ventricular cardiomyopathy (ARVC)
Dilated cardiomyopathy
Viral myocarditis
NetPath Pathway IL4 Signaling Pathway
IL5 Signaling Pathway
Reactome Non-integrin membrane-ECM interactions
Striated Muscle Contraction
WikiPathways Striated Muscle Contraction
Ectoderm Differentiation
Extracellular matrix organization
Arrhythmogenic Right Ventricular Cardiomyopathy
Muscle contraction
References
Ref 525046ClinicalTrials.gov (NCT02329769) Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD). U.S. National Institutes of Health.
Ref 532949Update on neuromuscular disorders in pediatric orthopaedics: Duchenne muscular dystrophy, myelomeningocele, and cerebral palsy. J Pediatr Orthop. 2014 Oct-Nov;34 Suppl 1:S44-8.

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