Properties (63)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
drug
|
| gptkbp:activeDuring |
eteplirsen
|
| gptkbp:chemicalFormula |
C20H26N2O5P
|
| gptkbp:clinicalTrials |
Phase 3
positive efficacy results NCT02310906 included in treatment protocols measured by functional tests multiple_sites_in_the_USA |
| gptkbp:collaborations |
various academic institutions
|
| gptkbp:community_service |
limited availability
dystrophin levels improvement in muscle function available_through_Sarepta_Therapeutics |
| gptkbp:contraindication |
serious allergic reactions
|
| gptkbp:customerFeedback |
generally positive
|
| gptkbp:date |
FDA_approved
|
| gptkbp:deathYear |
high cost
|
| gptkbp:developedBy |
gptkb:Sarepta_Therapeutics
|
| gptkbp:discontinued |
Yes
|
| gptkbp:dosageForm |
weekly
|
| gptkbp:drugInterdiction |
gptkb:antisense_oligonucleotide
increases dystrophin production half-life of approximately 4 hours |
| gptkbp:formulation |
sterile solution
|
| gptkbp:hasCompetitors |
other_DMD_therapies
|
| gptkbp:hasPopulation |
children and young adults
|
| gptkbp:healthcare |
ongoing clinical trials
important for prescribing supported_by_DMD_organizations |
| gptkbp:historicalResearch |
focused on combination therapies
|
| https://www.w3.org/2000/01/rdf-schema#label |
Eteplirsen
|
| gptkbp:impact |
long-term treatment
|
| gptkbp:insuranceAccepted |
varies by provider
|
| gptkbp:is_used_in |
ongoing evaluation
|
| gptkbp:isATypeOf |
162632-80-0
|
| gptkbp:lastProduced |
2016
|
| gptkbp:mandates |
gptkb:Duchenne_muscular_dystrophy_with_exon_51_skipping
|
| gptkbp:market |
2016
|
| gptkbp:marketedAs |
Exondys 51
|
| gptkbp:operates_in |
M09AX03
|
| gptkbp:patentStatus |
patented
|
| gptkbp:regulatoryCompliance |
conditional approval
|
| gptkbp:research |
public and private sources
|
| gptkbp:research_areas |
neuromuscular disorders
|
| gptkbp:researchAreas |
ongoing studies
|
| gptkbp:researchFocus |
genetic therapies
|
| gptkbp:researchInterest |
multiple peer-reviewed articles
with various biotech firms |
| gptkbp:route |
intravenous
subcutaneous |
| gptkbp:safetyFeatures |
generally well tolerated
|
| gptkbp:sideEffect |
fatigue
headache nausea injection site reactions |
| gptkbp:storage |
store at 2-8°C
|
| gptkbp:supplyChain |
managed_by_Sarepta_Therapeutics
|
| gptkbp:targets |
muscular dystrophy
patients_with_Duchenne_muscular_dystrophy |
| gptkbp:triggerType |
exon skipping
|
| gptkbp:usedFor |
gptkb:Duchenne_muscular_dystrophy
|
| gptkbp:weight |
409.5 g/mol
|