Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D3RT4A
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| Drug Name |
SRP-9003
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| Synonyms |
MYO-101
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|
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| Drug Type |
Gene therapy
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| Indication | Limb girdle muscular dystrophy [ICD-11: 8C70.4] | Phase 1/2 | [1] | |
| Company |
Sarepta Therapeutics
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Beta-sarcoglycan (SGCB) | Target Info | Replacement | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03652259) Gene Delivery Clinical Trial of SRP-9003 for Patients With LGMD2E (Beta-sarcoglycan Deficiency). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Sarepta Therapeutics. | |||
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