|
gptkbp:instance_of
|
gptkb:pharmaceuticals
|
|
gptkbp:affects
|
under investigation
|
|
gptkbp:approves
|
gptkb:2016
gptkb:European_Union
gptkb:United_States
|
|
gptkbp:availability
|
prescription only
|
|
gptkbp:brand
|
gptkb:Spinraza
|
|
gptkbp:clinical_trial
|
Phase 3
CHERISH
ENDEAR
NURTURE
SHINE
|
|
gptkbp:clinical_use
|
treatment of SMA
|
|
gptkbp:collaboration
|
academic institutions
biotechnology companies
|
|
gptkbp:developed_by
|
gptkb:Ionis_Pharmaceuticals
|
|
gptkbp:dosage_form
|
injection
|
|
gptkbp:duration
|
ongoing
|
|
gptkbp:financial_support
|
available
|
|
gptkbp:formulation
|
gptkb:Software_Solutions
|
|
gptkbp:funding
|
public and private
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
nusinersen
|
|
gptkbp:indication
|
gptkb:SMA_Type_1
SMA Type 2
SMA Type 3
|
|
gptkbp:ingredients
|
nusinersen sodium
|
|
gptkbp:invention
|
patented
|
|
gptkbp:market_launch
|
gptkb:2017
|
|
gptkbp:marketed_as
|
gptkb:Biogen
|
|
gptkbp:mechanism_of_action
|
antisense oligonucleotide
|
|
gptkbp:patient_population
|
gptkb:children
adults
infants
|
|
gptkbp:pharmacokinetics
|
dose-dependent
increased SMN protein levels
|
|
gptkbp:price
|
high
|
|
gptkbp:provides_guidance_on
|
recommended
|
|
gptkbp:regulatory_compliance
|
gptkb:FDA
gptkb:EMA
TGA
PMDA
|
|
gptkbp:research_areas
|
neuromuscular disorders
|
|
gptkbp:route_of_administration
|
intrathecal injection
|
|
gptkbp:safety_measures
|
required
|
|
gptkbp:side_effect
|
headache
nausea
positive
constipation
back pain
injection site reactions
|
|
gptkbp:storage
|
refrigerated
|
|
gptkbp:targets
|
gptkb:SMN2_gene
|
|
gptkbp:used_for
|
gptkb:muscular_dystrophy
|
|
gptkbp:bfsParent
|
gptkb:muscular_dystrophy
|
|
gptkbp:bfsLayer
|
5
|