Statements (64)
| Predicate | Object |
|---|---|
| gptkbp:instance_of |
gptkb:physicist
|
| gptkbp:bfsLayer |
3
|
| gptkbp:bfsParent |
gptkb:Novartis
|
| gptkbp:activities |
gene replacement therapy
|
| gptkbp:advocates_for |
supported by patient advocacy groups
|
| gptkbp:affects |
studied for long-term effects
|
| gptkbp:age |
up to 2 years old
|
| gptkbp:approves |
gptkb:legislation
gptkb:DJ gptkb:FDA gptkb:United_States 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 ST R1 VE |
| gptkbp:collaborations |
collaborations with research institutions
|
| gptkbp:developed_by |
gptkb:Novartis
|
| gptkbp:developer |
gptkb:Ave_Xis
|
| gptkbp:duration |
single dose
|
| gptkbp:effective_date |
gptkb:2019
|
| gptkbp:formulation |
liquid solution
|
| gptkbp:frequency |
once
|
| gptkbp:healthcare |
administered by healthcare professionals
available patient access programs |
| 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:manager |
intravenous
intravenous infusion |
| gptkbp:manufacturer |
gptkb:Novartis
|
| gptkbp:marketed_as |
gptkb:Zolgensma
Zolgensma brand name |
| gptkbp:marketing_strategy |
ongoing post-marketing studies
|
| gptkbp:population |
SMA patients
|
| gptkbp:price |
approximately $2.1 million
|
| gptkbp:products |
gptkb:drug
|
| gptkbp:provides_information_on |
included in treatment guidelines
|
| gptkbp:public_perception |
high public interest
|
| gptkbp:receives_funding_from |
significant investment in research
|
| gptkbp:regulatory_compliance |
approved
received multiple regulatory approvals |
| gptkbp:research_focus |
preclinical studies
|
| gptkbp:safety_features |
generally well tolerated
|
| gptkbp:serves |
gptkb:DJ
|
| gptkbp:side_effect |
potential liver toxicity
|
| gptkbp:student_enrollment |
diagnosed with SMA
|
| gptkbp:supply_chain |
complex supply chain management
|
| gptkbp:target_audience |
infants and young children
|
| gptkbp:targets |
gptkb:SMA_type_1
SM N1 gene |
| gptkbp:treatment |
high success rate
changed treatment paradigm for SMA improve survival significant impact on quality of life |
| gptkbp:type_of_insurance |
covered by some insurance plans
|