gptkbp:instance_of
|
gptkb:gene_therapy
|
gptkbp:administration_age
|
up to 2 years old
|
gptkbp:advocacy
|
supported by patient advocacy groups
|
gptkbp:affects
|
studied for long-term effects
|
gptkbp:approves
|
gptkb:European_Union
gptkb:FDA
gptkb:United_States
gptkb:muscular_dystrophy
May 2019
gene therapy for SMA
|
gptkbp:availability
|
available in multiple countries
|
gptkbp:capacity
|
5 m L
|
gptkbp:clinical_trial
|
Phase 3
positive outcomes
conducted in multiple countries
over 100 patients
STR1 VE
|
gptkbp:collaborations
|
collaborations with research institutions
|
gptkbp:developed_by
|
gptkb:Novartis
|
gptkbp:development
|
gptkb:Ave_Xis
|
gptkbp:disease_targeted
|
gptkb:SMA_type_1
|
gptkbp:duration
|
single dose
|
gptkbp:effective_date
|
gptkb:2019
|
gptkbp:eligibility
|
diagnosed with SMA
|
gptkbp:formulation
|
liquid solution
|
gptkbp:funding
|
significant investment in research
|
gptkbp:healthcare
|
administered by healthcare professionals
|
https://www.w3.org/2000/01/rdf-schema#label
|
Zolgensma
|
gptkbp:indication
|
children with spinal muscular atrophy
children under 2 years with SMA
|
gptkbp:ingredients
|
gptkb:onasemnogene_abeparvovec
gptkb:onasemnogene_abeparvovec-xioi
|
gptkbp:is_effective_against
|
improves motor function
|
gptkbp:is_monitored_by
|
requires follow-up monitoring
|
gptkbp:manufacturer
|
gptkb:Novartis
|
gptkbp:marketed_as
|
gptkb:Zolgensma
Zolgensma brand name
|
gptkbp:mechanism_of_action
|
gene replacement therapy
|
gptkbp:patient_access_programs
|
available patient access programs
|
gptkbp:patient_population
|
SMA patients
|
gptkbp:post-marketing_studies
|
ongoing post-marketing studies
|
gptkbp:price
|
approximately $2.1 million
|
gptkbp:product_type
|
gptkb:pharmaceuticals
|
gptkbp:provides_guidance_on
|
included in treatment guidelines
|
gptkbp:public_perception
|
high public interest
|
gptkbp:regulatory_compliance
|
approved
received multiple regulatory approvals
|
gptkbp:research_phase
|
preclinical studies
|
gptkbp:route_of_administration
|
intravenous
intravenous infusion
|
gptkbp:safety_features
|
generally well tolerated
|
gptkbp:serves
|
gptkb:muscular_dystrophy
|
gptkbp:service_frequency
|
once
|
gptkbp:side_effect
|
potential liver toxicity
|
gptkbp:supply_chain
|
complex supply chain management
|
gptkbp:target_audience
|
infants and young children
|
gptkbp:target_gene
|
gptkb:SMN1_gene
|
gptkbp:treatment
|
high success rate
improve survival
|
gptkbp:treatment_impact
|
significant impact on quality of life
|
gptkbp:treatment_paradigm
|
changed treatment paradigm for SMA
|
gptkbp:type_of_insurance
|
covered by some insurance plans
|
gptkbp:bfsParent
|
gptkb:Novartis
|
gptkbp:bfsLayer
|
4
|