Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D9M8FK
|
|||
| Drug Name |
SRP-9001
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Duchenne dystrophy [ICD-11: 8C70; ICD-10: G71.0] | Phase 2 | [1] | |
| Company |
Sarepta Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Dystrophin (DMD) | Target Info | Replacement | [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 (NCT03769116) A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Sarepta Therapeutics. | |||
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