|
gptkbp:instance_of
|
gptkb:pharmaceuticals
|
|
gptkbp:access_programs
|
available for patients in need
|
|
gptkbp:accessories
|
none reported
|
|
gptkbp:approves
|
gptkb:FDA
|
|
gptkbp:atccode
|
M09 AX03
|
|
gptkbp:availability
|
available in multiple countries
|
|
gptkbp:bioavailability
|
high
|
|
gptkbp:casnumber
|
1195760-78-6
|
|
gptkbp:chemical_formula
|
C20 H24 N4 O3 S
|
|
gptkbp:clinical_trial
|
ongoing
Phase 3
|
|
gptkbp:collaborations
|
with academic institutions
|
|
gptkbp:community_support
|
active patient advocacy groups
|
|
gptkbp:contraindication
|
none known
|
|
gptkbp:developed_by
|
gptkb:Roche
|
|
gptkbp:dosage_form
|
gptkb:liquid
once daily
|
|
gptkbp:drug_interactions
|
none significant
|
|
gptkbp:duration
|
long-term therapy
|
|
gptkbp:effective_date
|
approved
August 7, 2020
|
|
gptkbp:excretion
|
urine
|
|
gptkbp:feedback
|
generally positive
|
|
gptkbp:financial_support
|
available
|
|
gptkbp:formulation
|
suspension
|
|
gptkbp:funding
|
funded by Roche
|
|
gptkbp:future_prospects
|
focused on long-term effects
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Risdiplam
|
|
gptkbp:indication
|
treatment of SMA
|
|
gptkbp:invention
|
patented
|
|
gptkbp:is_effective_against
|
improves motor function
|
|
gptkbp:is_monitored_by
|
liver function tests
|
|
gptkbp:lifespan
|
approximately 12 hours
|
|
gptkbp:manufacturer
|
gptkb:Genentech
|
|
gptkbp:market_launch
|
gptkb:2020
|
|
gptkbp:marketed_as
|
gptkb:Evrysdi
|
|
gptkbp:mechanism_of_action
|
SMN2 gene splicing
|
|
gptkbp:patient_education
|
important for understanding treatment
|
|
gptkbp:patient_population
|
children and adults
|
|
gptkbp:pharmacokinetics
|
linear
increases SMN protein levels
|
|
gptkbp:price
|
varies by region
|
|
gptkbp:provides_guidance_on
|
included in SMA treatment guidelines
|
|
gptkbp:publication
|
numerous studies published
|
|
gptkbp:regulatory_compliance
|
approved by EMA
|
|
gptkbp:research_status
|
active research
|
|
gptkbp:route_of_administration
|
oral
|
|
gptkbp:safety_features
|
generally well tolerated
|
|
gptkbp:side_effect
|
fatigue
nausea
fever
diarrhea
rash
monitor for signs of liver toxicity
|
|
gptkbp:storage
|
room temperature
|
|
gptkbp:supply_chain
|
commercially available
|
|
gptkbp:targets
|
gptkb:SMN2
|
|
gptkbp:treatment
|
positive results in trials
|
|
gptkbp:type_of_care
|
important for efficacy
|
|
gptkbp:type_of_insurance
|
varies by plan
|
|
gptkbp:used_for
|
gptkb:muscular_dystrophy
|
|
gptkbp:weight
|
396.49 g/mol
|
|
gptkbp:bfsParent
|
gptkb:SMA_type_1
|
|
gptkbp:bfsLayer
|
6
|