Statements (22)
| Predicate | Object | 
|---|---|
| gptkbp:instanceOf | gptkb:drug | 
| gptkbp:approvalYear | 2019 | 
| gptkbp:approvedBy | gptkb:FDA | 
| gptkbp:ATCCode | M09AX10 | 
| gptkbp:CASNumber | 1446729-34-7 | 
| gptkbp:developedBy | gptkb:Sarepta_Therapeutics | 
| gptkbp:exonTargeted | exon 53 | 
| gptkbp:form | solution for injection | 
| gptkbp:genericName | golodirsen | 
| gptkbp:hasMolecularFormula | C615H941N173O176P30 | 
| gptkbp:indication | gptkb:Duchenne_muscular_dystrophy | 
| gptkbp:legalStatus | prescription only | 
| gptkbp:mechanismOfAction | exon skipping | 
| gptkbp:orphanDrugStatus | yes | 
| gptkbp:pregnancyCategory | not assigned | 
| gptkbp:routeOfAdministration | intravenous | 
| gptkbp:synonym | SRP-4053 | 
| gptkbp:targetGene | gptkb:DMD_gene | 
| gptkbp:UNII | 6H4G63K35Q | 
| gptkbp:bfsParent | gptkb:Sarepta_Therapeutics | 
| gptkbp:bfsLayer | 7 | 
| https://www.w3.org/2000/01/rdf-schema#label | Vyondys 53 |