Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D05DFV
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| Former ID |
DIB004035
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| Drug Name |
PRO-044
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| Synonyms |
Exon-44 skipping antisense oligonucleotide (Duchenne muscular dystrophy), Prosensa
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|
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| Drug Type |
Antisense drug
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| Indication | Duchenne dystrophy [ICD-11: 8C70; ICD-10: G71.0] | Phase 2 | [1] | |
| Company |
Leiden University Medical Center
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Dystrophin messenger RNA (DMD mRNA) | Target Info | . | [2] |
| 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 | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT02329769) Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD). U.S. National Institutes of Health. | |||
| REF 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. | |||
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