|
gptkbp:instance_of
|
gptkb:pharmaceuticals
|
|
gptkbp:affects
|
under investigation
|
|
gptkbp:approves
|
gptkb:FDA
|
|
gptkbp:brand
|
Vyondys 53
|
|
gptkbp:chemical_formula
|
C202 H267 N29 O41 P
|
|
gptkbp:clinical_trial
|
gptkb:United_States
gptkb:Duchenne_muscular_dystrophy
gptkb:Sarepta_Therapeutics
Phase 3
published
positive efficacy
|
|
gptkbp:clinical_trial_endpoints
|
safety assessment
biomarker analysis
muscle function improvement
|
|
gptkbp:collaborations
|
academic institutions
|
|
gptkbp:community_health
|
provided
|
|
gptkbp:developed_by
|
gptkb:Sarepta_Therapeutics
|
|
gptkbp:dosage_form
|
weekly intravenous infusion
|
|
gptkbp:drug_interactions
|
none reported
|
|
gptkbp:duration
|
ongoing
|
|
gptkbp:effective_date
|
gptkb:2019
|
|
gptkbp:eligibility
|
genetic testing
age criteria
Duchenne muscular dystrophy diagnosis
|
|
gptkbp:financial_support
|
available
|
|
gptkbp:formulation
|
sterile solution
|
|
gptkbp:funding
|
public and private sources
|
|
gptkbp:grants
|
awarded
|
|
gptkbp:healthcare
|
gptkb:true
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Golodirsen
|
|
gptkbp:indication
|
muscle weakness
|
|
gptkbp:ingredients
|
oligonucleotide
|
|
gptkbp:invention
|
patented
|
|
gptkbp:market_authorization
|
granted
|
|
gptkbp:marketed_as
|
gptkb:United_States
|
|
gptkbp:mechanism_of_action
|
exon skipping
|
|
gptkbp:patient_population
|
Duchenne muscular dystrophy patients
|
|
gptkbp:pharmacokinetics
|
variable absorption
antisense oligonucleotide therapy
|
|
gptkbp:provides_guidance_on
|
available
followed
|
|
gptkbp:publication
|
numerous
|
|
gptkbp:regulatory_compliance
|
approved
|
|
gptkbp:research_areas
|
genetic disorders
neuromuscular diseases
|
|
gptkbp:route_of_administration
|
intravenous
infusion
|
|
gptkbp:safety_features
|
monitored
|
|
gptkbp:safety_measures
|
ongoing
|
|
gptkbp:service_frequency
|
weekly
|
|
gptkbp:short_term_effects
|
assessed
|
|
gptkbp:side_effect
|
fatigue
headache
nausea
fever
vomiting
diarrhea
injection site reactions
muscle cramps
hypersensitivity reactions
increased liver enzymes
|
|
gptkbp:target_audience
|
children and young adults
|
|
gptkbp:targets
|
gptkb:Duchenne_muscular_dystrophy
|
|
gptkbp:treatment
|
monitored
evaluated
|
|
gptkbp:type_of_care
|
monitored
|
|
gptkbp:weight
|
3300.5 g/mol
|
|
gptkbp:bfsParent
|
gptkb:Sarepta_Therapeutics
|
|
gptkbp:bfsLayer
|
5
|