gptkbp:instance_of
|
gptkb:pharmaceuticals
|
gptkbp:approves
|
gptkb:2020
gptkb:FDA
|
gptkbp:atccode
|
M09 AX03
|
gptkbp:average_temperature
|
room temperature
|
gptkbp:bioavailability
|
high
|
gptkbp:brand
|
gptkb:Evrysdi
|
gptkbp:casnumber
|
1446320-73-0
|
gptkbp:chemical_formula
|
C20 H25 N3 O2
|
gptkbp:class
|
SMN2 splicing modifier
|
gptkbp:clinical_trial
|
Phase 3
FIREFISH
SUNFISH
RAINBOW
|
gptkbp:composed_by
|
gptkb:chemical_compound
|
gptkbp:contraindication
|
severe liver impairment
hypersensitivity to risdiplam
|
gptkbp:developed_by
|
gptkb:Roche
|
gptkbp:dosage_form
|
gptkb:Software_Solutions
|
gptkbp:excretion
|
urine
|
gptkbp:formulation
|
oral solution
|
https://www.w3.org/2000/01/rdf-schema#label
|
risdiplam
|
gptkbp:indication
|
treatment of spinal muscular atrophy
|
gptkbp:interacts_with
|
other medications affecting liver enzymes
|
gptkbp:invention
|
patented
|
gptkbp:label
|
prescribing information
|
gptkbp:lifespan
|
approximately 12 hours
|
gptkbp:marketed_as
|
gptkb:Australia
gptkb:Canada
gptkb:European_Union
gptkb:Japan
gptkb:United_States
|
gptkbp:mechanism_of_action
|
SMN2 gene splicing
|
gptkbp:packaging
|
gptkb:bottle
|
gptkbp:patient_population
|
pediatric patients
adult patients
|
gptkbp:research_focus
|
gptkb:gene_therapy
neuromuscular diseases
clinical efficacy
safety profile
SMN protein levels
|
gptkbp:route_of_administration
|
oral
|
gptkbp:side_effect
|
fatigue
nausea
fever
vomiting
diarrhea
|
gptkbp:targets
|
survival motor neuron protein
|
gptkbp:used_for
|
gptkb:muscular_dystrophy
|
gptkbp:weight
|
337.44 g/mol
|
gptkbp:bfsParent
|
gptkb:muscular_dystrophy
|
gptkbp:bfsLayer
|
5
|