Statements (29)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gptkb:drug
gene therapy |
| gptkbp:age |
4 to 5 years
|
| gptkbp:approvalYear |
2023-06-22
|
| gptkbp:approvedBy |
gptkb:FDA
|
| gptkbp:ATCCode |
M09AX14
|
| gptkbp:blackBoxWarning |
acute serious liver injury
|
| gptkbp:countryOfOperation |
gptkb:United_States
|
| gptkbp:developedBy |
gptkb:Sarepta_Therapeutics
|
| gptkbp:form |
solution for infusion
|
| gptkbp:genericName |
delandistrogene moxeparvovec
|
| https://www.w3.org/2000/01/rdf-schema#label |
Elevidys
|
| gptkbp:indication |
gptkb:Duchenne_muscular_dystrophy
|
| gptkbp:legalStatus |
prescription only
|
| gptkbp:mechanismOfAction |
delivers micro-dystrophin gene
|
| gptkbp:orphanDrugStatus |
yes
|
| gptkbp:pregnancyCategory |
not established
|
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:sideEffect |
fever
nausea vomiting increased liver enzymes acute liver injury decreased platelet count |
| gptkbp:storage |
2°C to 8°C
|
| gptkbp:targetGene |
gptkb:DMD_gene
|
| gptkbp:vectorFor |
AAVrh74 viral vector
|
| gptkbp:bfsParent |
gptkb:Sarepta_Therapeutics
|
| gptkbp:bfsLayer |
7
|