Statements (94)
| Predicate | Object |
|---|---|
| gptkbp:instance_of |
gptkb:gene_therapy
|
| gptkbp:administration_age |
less than 2 years
|
| gptkbp:advocacy |
strong community support
|
| gptkbp:approves |
gptkb:FDA
gptkb:United_States |
| gptkbp:availability |
limited
|
| gptkbp:biologic_type |
gptkb:recombinant_adeno-associated_virus
|
| gptkbp:clinical_trial |
gptkb:Ave_Xis
randomized controlled trials multiple sites published in journals secured from various sources positive results Phase 1/2 criteria defined multiple phases conducted reported to regulatory agencies encouraged for eligible patients |
| gptkbp:clinical_trial_phase2 |
ongoing
|
| gptkbp:clinical_trial_results_publication |
peer-reviewed journals
|
| gptkbp:collaborations |
with academic institutions
with pharmaceutical companies |
| gptkbp:community_health |
important for effective treatment
influences treatment availability |
| gptkbp:developed_by |
gptkb:Ave_Xis
|
| gptkbp:dosing_weight |
up to 21 kg
|
| gptkbp:duration |
lifelong benefits
|
| gptkbp:effective_date |
gptkb:2019
|
| gptkbp:eligibility |
diagnosed with SMA
|
| gptkbp:feedback |
collected for improvement
|
| gptkbp:financial_support |
available
|
| gptkbp:funding |
public and private sources
|
| gptkbp:future_prospects |
focused on long-term effects
|
| gptkbp:healthcare |
high initial investment
|
| gptkbp:healthcare_provider_guidance |
essential for treatment success
|
| gptkbp:healthcare_system_impact |
significant due to cost
|
| https://www.w3.org/2000/01/rdf-schema#label |
Onasemnogene abeparvovec
|
| gptkbp:indication |
gptkb:SMA_Type_1
|
| gptkbp:is_effective_against |
improves motor function
|
| gptkbp:is_monitored_by |
required for safety
required post-treatment |
| gptkbp:long_term_effectiveness |
under investigation
|
| gptkbp:manufacturer |
gptkb:Novartis
|
| gptkbp:market |
ongoing
|
| gptkbp:marketed_as |
gptkb:Zolgensma
|
| gptkbp:mechanism_of_action |
gene replacement therapy
delivers functional SMN gene |
| gptkbp:patient_advocacy_groups |
support treatment access
|
| gptkbp:patient_education |
critical for understanding treatment
|
| gptkbp:patient_outreach |
important for awareness
|
| gptkbp:patient_population |
infants and young children
varied across studies |
| gptkbp:patient_support_resources |
available for families
|
| gptkbp:price |
high
|
| gptkbp:provides_guidance_on |
updated regularly
limited in some countries developed for use |
| gptkbp:publication |
numerous studies
|
| gptkbp:recruitment |
based on genetic testing
|
| gptkbp:regulatory_compliance |
approved
based on clinical trial data |
| gptkbp:research_areas |
genetic disorders
|
| gptkbp:research_focus |
neuromuscular disorders
|
| gptkbp:route_of_administration |
intravenous
IV infusion |
| gptkbp:safety |
monitored closely post-treatment
|
| gptkbp:safety_features |
generally well tolerated
|
| gptkbp:serves |
gptkb:muscular_dystrophy
|
| gptkbp:service_frequency |
single dose
|
| gptkbp:side_effect |
liver enzyme elevation
significant improvement |
| gptkbp:supply_chain |
complex and regulated
|
| gptkbp:target_gene |
gptkb:SMN1
|
| gptkbp:training |
necessary for administration
|
| gptkbp:treatment |
assessed in studies
monitored long-term standardized guidelines continues to be evaluated improve survival and motor function ongoing in multiple centers |
| gptkbp:treatment_access_challenges |
exist in some regions
|
| gptkbp:treatment_impact |
significant on quality of life
|
| gptkbp:treatment_innovation |
represents a breakthrough
|
| gptkbp:treatment_limitations |
not suitable for all patients
|
| gptkbp:treatment_monitoring_requirements |
defined by clinical guidelines
|
| gptkbp:treatment_outcomes_research |
ongoing to assess long-term effects
|
| gptkbp:treatment_research_collaboration |
with academic institutions and industry
|
| gptkbp:treatment_research_funding |
critical for advancement
|
| gptkbp:type |
AAV-based gene therapy
|
| gptkbp:type_of_care |
key for treatment success
multidisciplinary approach required |
| gptkbp:type_of_insurance |
varies by region
|
| gptkbp:bfsParent |
gptkb:SMA_type_1
|
| gptkbp:bfsLayer |
6
|