|
gptkbp:instanceOf
|
drug
|
|
gptkbp:activeDuring
|
gptkb:Golodirsen
|
|
gptkbp:administrativeDivision
|
weekly
|
|
gptkbp:approves
|
gptkb:FDA
|
|
gptkbp:availability
|
prescription only
|
|
gptkbp:chemicalFormula
|
C202H267N29O49P
|
|
gptkbp:clinicalTrials
|
gptkb:Canada
gptkb:United_States
gptkb:Sarepta_Therapeutics
Europe
Phase 3
positive outcomes
available for healthcare providers
Phase 1, Phase 2
|
|
gptkbp:community_service
|
offered by manufacturer
|
|
gptkbp:contraindication
|
serious allergic reactions
|
|
gptkbp:customerFeedback
|
generally positive
|
|
gptkbp:developedBy
|
gptkb:Sarepta_Therapeutics
|
|
gptkbp:discontinued
|
yes
|
|
gptkbp:dosageForm
|
based on body weight
|
|
gptkbp:drugInterdiction
|
available
genetic medicine
limited data available
|
|
gptkbp:firstAppearance
|
December 2019
|
|
gptkbp:formulation
|
sterile solution
|
|
gptkbp:hasCompetitors
|
other_DMD_therapies
|
|
gptkbp:hasPopulation
|
children and adults
|
|
gptkbp:healthcare
|
specific genetic mutations
supported_by_DMD_organizations
provided_by_Sarepta_Therapeutics
|
|
gptkbp:historicalResearch
|
exploring long-term effects
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Golodirsen
|
|
gptkbp:inventorResidence
|
clinical trial data
|
|
gptkbp:is_monitored_by
|
regular blood tests
|
|
gptkbp:mandates
|
gptkb:Duchenne_muscular_dystrophy_with_exon_53_skipping
|
|
gptkbp:market
|
2020
|
|
gptkbp:marketedAs
|
Vyondys 53
|
|
gptkbp:patentStatus
|
patented
|
|
gptkbp:regulatoryBody
|
gptkb:FDA
|
|
gptkbp:regulatoryCompliance
|
conditional approval
|
|
gptkbp:research
|
public and private sources
|
|
gptkbp:research_areas
|
neuromuscular disorders
|
|
gptkbp:researchAreas
|
ongoing studies
|
|
gptkbp:researchInterest
|
with academic institutions
|
|
gptkbp:route
|
intravenous
|
|
gptkbp:safetyFeatures
|
generally well tolerated
|
|
gptkbp:shelfLife
|
24 months
|
|
gptkbp:sideEffect
|
fatigue
headache
nausea
fever
injection site reactions
|
|
gptkbp:storage
|
refrigerated
|
|
gptkbp:targets
|
gptkb:Duchenne_muscular_dystrophy
|
|
gptkbp:triggerType
|
gptkb:antisense_oligonucleotide
|
|
gptkbp:type
|
exon skipping therapy
|