|
gptkbp:instance_of
|
gptkb:gene_therapy
|
|
gptkbp:administration_age
|
less than 2 years old
|
|
gptkbp:administration_setting
|
gptkb:hospital
|
|
gptkbp:advocacy
|
support groups
|
|
gptkbp:approves
|
gptkb:2019
gptkb:FDA
|
|
gptkbp:biologic_type
|
gptkb:virus
|
|
gptkbp:clinical_trial
|
gptkb:United_States
gptkb:children
Phase 2
Phase 3
multiple phases
Phase 1/2
open-label study
positive efficacy
improved motor function
ongoing recruitment
|
|
gptkbp:collaborations
|
academic institutions
|
|
gptkbp:developed_by
|
gptkb:Ave_Xis
|
|
gptkbp:disease_target
|
gptkb:spinal_muscular_atrophy_type_1
|
|
gptkbp:dosage_form
|
single-dose treatment
|
|
gptkbp:duration
|
long-term efficacy
|
|
gptkbp:eligibility
|
diagnosed with SMA
|
|
gptkbp:feedback
|
positive experiences
|
|
gptkbp:financial_support
|
financial assistance programs
|
|
gptkbp:funding
|
gptkb:pharmaceuticals
|
|
gptkbp:genetic_studies
|
SMN protein production
|
|
gptkbp:healthcare
|
specialized clinics
high treatment costs
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
onasemnogene abeparvovec
|
|
gptkbp:impact
|
significant advancements in SMA treatment
|
|
gptkbp:indication
|
children with spinal muscular atrophy
|
|
gptkbp:invention
|
gptkb:Ave_Xis
|
|
gptkbp:is_monitored_by
|
post-treatment follow-up
|
|
gptkbp:long_term_follow_up
|
ongoing studies
|
|
gptkbp:marketed_as
|
gptkb:Zolgensma
|
|
gptkbp:mechanism_of_action
|
gene replacement therapy
|
|
gptkbp:patient_population
|
infants and young children
|
|
gptkbp:price
|
high
|
|
gptkbp:provides_guidance_on
|
limited availability
evidence-based recommendations
recommended for early intervention
|
|
gptkbp:publication
|
peer-reviewed journals
|
|
gptkbp:regulatory_compliance
|
gptkb:European_Medicines_Agency
approved
|
|
gptkbp:research_areas
|
neuromuscular disorders
|
|
gptkbp:research_focus
|
gene therapy advancements
|
|
gptkbp:route_of_administration
|
intravenous
|
|
gptkbp:safety_features
|
generally well tolerated
|
|
gptkbp:service_frequency
|
once in a lifetime
|
|
gptkbp:side_effect
|
liver enzyme elevation
|
|
gptkbp:sponsor
|
gptkb:Novartis
|
|
gptkbp:target_audience
|
pediatric patients
|
|
gptkbp:targets
|
gptkb:SMN1_gene
|
|
gptkbp:treatment
|
improved quality of life
high success rate
ongoing clinical studies
single intravenous infusion
curative therapy
increase survival
restores SMN protein levels
significant improvement
|
|
gptkbp:treatment_limitations
|
age restrictions
|
|
gptkbp:type_of_insurance
|
varies by plan
|
|
gptkbp:used_for
|
treatment of spinal muscular atrophy
|
|
gptkbp:bfsParent
|
gptkb:Zolgensma
gptkb:muscular_dystrophy
|
|
gptkbp:bfsLayer
|
5
|