Properties (65)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
drug
|
| gptkbp:activeDuring |
nusinersen
|
| gptkbp:administrativeDivision |
every 4 months after loading doses
injection into the spinal canal |
| gptkbp:advocacy |
supporting_SMA_awareness
|
| gptkbp:approves |
gptkb:FDA
|
| gptkbp:clinicalTrials |
multiple countries
Phase 3 improved survival rates specific genetic mutations available for financial assistance completed and ongoing trials positive outcomes in motor function recommended_for_SMA_treatment treatment_of_SMA |
| gptkbp:community_service |
available
|
| gptkbp:contraindication |
seizures
respiratory issues serious infections hypersensitivity reactions |
| gptkbp:customerFeedback |
generally positive
|
| gptkbp:deathYear |
approximately $750,000
|
| gptkbp:dosageForm |
solution
initial loading doses followed by maintenance doses |
| gptkbp:drugInterdiction |
none significant reported
neuromuscular agent |
| gptkbp:globalPresence |
widely available in developed countries
|
| gptkbp:hasCompetitors |
other_SMA_therapies
|
| gptkbp:hasPopulation |
infants, children, adults
|
| gptkbp:healthcare |
important for efficacy
important for understanding treatment necessary for administration important for treatment access significant burden on healthcare systems diagnosed_with_SMA |
| https://www.w3.org/2000/01/rdf-schema#label |
Spinraza
|
| gptkbp:impact |
improves motor function
|
| gptkbp:is_monitored_by |
regular follow-up assessments
|
| gptkbp:is_used_in |
ongoing evaluation
|
| gptkbp:lastProduced |
2016
|
| gptkbp:manufacturer |
gptkb:Ionis_Pharmaceuticals
|
| gptkbp:marketedAs |
brand name
|
| gptkbp:notableFeature |
Zolgensma
Evrysdi |
| gptkbp:patentExpiration |
2028
|
| gptkbp:regulatoryCompliance |
approved in multiple countries
based on clinical trial data |
| gptkbp:research |
public and private sectors
|
| gptkbp:researchAreas |
ongoing studies
|
| gptkbp:researchFocus |
long-term efficacy and safety
|
| gptkbp:researchInterest |
academic institutions
numerous peer-reviewed articles |
| gptkbp:route |
intrathecal injection
|
| gptkbp:safetyFeatures |
risk of thrombocytopenia
|
| gptkbp:sideEffect |
headache
nausea constipation back pain injection site reactions |
| gptkbp:storage |
refrigerated
|
| gptkbp:supplyChain |
managed by manufacturer
|
| gptkbp:targets |
children_and_adults_with_SMA
|
| gptkbp:triggerType |
gptkb:antisense_oligonucleotide
|
| gptkbp:typeOfInsurance |
varies by region
|
| gptkbp:usedFor |
spinal muscular atrophy
|