|
gptkbp:instanceOf
|
gene therapy
|
|
gptkbp:acquiredBy
|
gptkb:Novartis
|
|
gptkbp:administeredBy
|
single-dose infusion
|
|
gptkbp:alternativeTreatment
|
gptkb:Evrysdi
gptkb:Spinraza
|
|
gptkbp:approvedBy
|
gptkb:FDA
gptkb:EMA
2019-05-24
|
|
gptkbp:ATCCode
|
gptkb:M09AX16
|
|
gptkbp:blackBoxWarning
|
acute serious liver injury
|
|
gptkbp:clinicalTrialPhase
|
Phase 3
|
|
gptkbp:cost
|
over $2 million per dose
|
|
gptkbp:countryOfOrigin
|
gptkb:United_States
|
|
gptkbp:developedBy
|
gptkb:AveXis
|
|
gptkbp:geneDelivered
|
gptkb:SMN1
|
|
gptkbp:genericName
|
gptkb:onasemnogene_abeparvovec
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Zolgensma
|
|
gptkbp:indication
|
pediatric patients under 2 years old with SMA
|
|
gptkbp:manufacturer
|
gptkb:Novartis
|
|
gptkbp:mechanismOfAction
|
delivers functional copy of SMN1 gene
|
|
gptkbp:orphanDrugStatus
|
yes
|
|
gptkbp:pregnancyCategory
|
not established
|
|
gptkbp:prescriptionStatus
|
prescription only
|
|
gptkbp:routeOfAdministration
|
intravenous
|
|
gptkbp:sideEffect
|
vomiting
elevated liver enzymes
thrombocytopenia
acute liver injury
|
|
gptkbp:storage
|
frozen
|
|
gptkbp:treatment
|
spinal muscular atrophy
|
|
gptkbp:vectorFor
|
gptkb:adeno-associated_virus_serotype_9_(AAV9)
|
|
gptkbp:bfsParent
|
gptkb:Novartis
|
|
gptkbp:bfsLayer
|
4
|