Statements (66)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:advocacy |
support treatment access
|
| gptkbp:approves |
gptkb:United_States
|
| gptkbp:caseTypes |
recombinant adeno-associated virus
|
| gptkbp:clinicalTrials |
Novartis
diverse demographics multiple countries Phase 3 randomized controlled trials updated regularly positive outcomes monitored over time demonstrated efficacy improved survival rates monitored by regulatory agencies completed and ongoing trials NCT02122952 cost and access issues recommended for early treatment |
| gptkbp:collaborations |
gptkb:University_of_Pennsylvania
|
| gptkbp:community |
reduces healthcare costs
|
| gptkbp:community_service |
available
|
| gptkbp:contraindication |
liver enzyme elevation
|
| gptkbp:customerFeedback |
generally positive
|
| gptkbp:developedBy |
AveXis
|
| gptkbp:diseaseResistance |
varies by patient
restores SMN protein |
| gptkbp:dosageForm |
single-dose
|
| gptkbp:evaluates |
improves motor function
|
| gptkbp:geneticDiversity |
AAV9_vector
|
| gptkbp:globalPresence |
available in select countries
|
| gptkbp:hasPopulation |
infants and young children
|
| gptkbp:healthcare |
important for treatment success
required post-treatment critical for awareness essential for understanding treatment. diagnosed_with_SMA_type_1 |
| gptkbp:historicalResearch |
focused on long-term effects
|
| https://www.w3.org/2000/01/rdf-schema#label |
onasemnogene abeparvovec
|
| gptkbp:impact |
limited in some regions
lifelong benefits |
| gptkbp:insuranceAccepted |
varies by region
|
| gptkbp:lastProduced |
2019
|
| gptkbp:lengthOverall |
under investigation
|
| gptkbp:mandates |
children with spinal muscular atrophy
|
| gptkbp:market |
May 2019
|
| gptkbp:marketedAs |
Zolgensma
|
| gptkbp:marketSegment |
ongoing
|
| gptkbp:offers |
approximately $2.1 million
|
| gptkbp:oversees |
under 2 years old
|
| gptkbp:patentStatus |
patented
|
| gptkbp:regulatoryCompliance |
navigated successfully
FDA_approved EMA_approved |
| gptkbp:research |
public and private sources
government and private sector |
| gptkbp:researchFocus |
neuromuscular disorders
|
| gptkbp:researchInterest |
numerous studies published
multiple academic institutions |
| gptkbp:route |
intravenous
|
| gptkbp:safetyFeatures |
generally well tolerated
|
| gptkbp:serves |
spinal muscular atrophy
|
| gptkbp:supplyChain |
managed_by_Novartis
|
| gptkbp:targets |
SMN1
|
| gptkbp:triggerType |
gene replacement therapy
|
| gptkbp:type |
one-time treatment
|