|
gptkbp:instance_of
|
gptkb:pharmaceuticals
|
|
gptkbp:administered_by
|
healthcare professional
|
|
gptkbp:advocacy
|
supported by SMA organizations
|
|
gptkbp:approves
|
gptkb:European_Union
gptkb:FDA
gptkb:United_States
|
|
gptkbp:availability
|
available in many countries
|
|
gptkbp:average_temperature
|
2 to 8 degrees Celsius
|
|
gptkbp:clinical_trial
|
Phase 3
CHERISH
ENDEAR
NURTURE
SHINE
|
|
gptkbp:cost_effectiveness
|
evaluated in studies
|
|
gptkbp:cost_per_year
|
approximately $750,000
|
|
gptkbp:developed_by
|
gptkb:Ionis_Pharmaceuticals
|
|
gptkbp:dosage_form
|
gptkb:Software_Solutions
|
|
gptkbp:effective_date
|
gptkb:2016
|
|
gptkbp:financial_support
|
available
|
|
gptkbp:frequency
|
every 4 months after loading doses
|
|
gptkbp:funding
|
public and private funding
|
|
gptkbp:healthcare
|
may vary by region
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Spinraza
|
|
gptkbp:indication
|
children and adults with spinal muscular atrophy
|
|
gptkbp:ingredients
|
gptkb:nusinersen
|
|
gptkbp:invention
|
patented
|
|
gptkbp:is_effective_against
|
improves motor function
delays disease progression
increases survival
|
|
gptkbp:is_monitored_by
|
regular follow-up visits
|
|
gptkbp:launch_date
|
gptkb:2017
|
|
gptkbp:loading_doses
|
initial 4 doses
|
|
gptkbp:long_term_outcomes
|
ongoing research
|
|
gptkbp:manufacturer
|
gptkb:Biogen_Idec
|
|
gptkbp:marketed_as
|
gptkb:Biogen
|
|
gptkbp:mechanism_of_action
|
antisense oligonucleotide
|
|
gptkbp:orphan_drug_status
|
yes
|
|
gptkbp:patient_education
|
important for treatment adherence
|
|
gptkbp:patient_population
|
gptkb:children
adults
infants
|
|
gptkbp:provides_guidance_on
|
included in SMA treatment guidelines
|
|
gptkbp:publication
|
numerous peer-reviewed articles
|
|
gptkbp:regulatory_compliance
|
conditional approval
|
|
gptkbp:research_focus
|
gptkb:muscular_dystrophy
|
|
gptkbp:risk_factor
|
injection site reactions
thrombocytopenia
serious infections
hypersensitivity reactions
|
|
gptkbp:route_of_administration
|
intrathecal injection
|
|
gptkbp:serves
|
gptkb:muscular_dystrophy
|
|
gptkbp:side_effect
|
headache
nausea
constipation
back pain
respiratory infections
|
|
gptkbp:type_of_insurance
|
varies by plan
|
|
gptkbp:bfsParent
|
gptkb:Biogen_MA_Inc.
gptkb:Nusinersen
gptkb:Ionis_Pharmaceuticals
|
|
gptkbp:bfsLayer
|
5
|