|
gptkbp:instance_of
|
gptkb:Biology
|
|
gptkbp:approves
|
gptkb:European_Union
gptkb:United_States
|
|
gptkbp:clinical_trial
|
Phase 3
NCT00158600
NCT00158613
NCT00158626
NCT00158639
NCT00158652
|
|
gptkbp:clinical_use
|
gptkb:Biology
|
|
gptkbp:contraindication
|
severe respiratory disease
hypersensitivity to alglucosidase alfa
|
|
gptkbp:dosage_form
|
solution for infusion
|
|
gptkbp:duration
|
lifelong
|
|
gptkbp:effective_date
|
2006-04-28
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Myozyme
|
|
gptkbp:indication
|
gptkb:infantile-onset_Pompe_disease
gptkb:late-onset_Pompe_disease
|
|
gptkbp:ingredients
|
gptkb:alglucosidase_alfa
|
|
gptkbp:is_monitored_by
|
required
|
|
gptkbp:manufacturer
|
gptkb:Genzyme
|
|
gptkbp:market_position
|
available
|
|
gptkbp:mechanism_of_action
|
replaces deficient enzyme
|
|
gptkbp:orphan_drug_status
|
yes
|
|
gptkbp:packaging
|
vial
|
|
gptkbp:patient_education
|
importance of adherence
need for regular follow-up
recognition of side effects
|
|
gptkbp:patient_population
|
gptkb:children
adults
|
|
gptkbp:pharmacokinetics
|
half-life of 2-3 hours
improves muscle function
increases glycogen breakdown
reduces glycogen accumulation
|
|
gptkbp:provides_guidance_on
|
recommended for early diagnosis
recommended for newborn screening
|
|
gptkbp:research_focus
|
combination therapies
quality of life improvements
new formulations
long-term efficacy
|
|
gptkbp:route_of_administration
|
IV
intravenous infusion
|
|
gptkbp:service_frequency
|
every 2 weeks
|
|
gptkbp:shelf_life
|
24 months
|
|
gptkbp:side_effect
|
headache
muscle pain
nausea
fever
vomiting
urticaria
respiratory infections
anaphylaxis
hypotension
infusion-related reactions
|
|
gptkbp:storage
|
refrigerated
|
|
gptkbp:structure
|
glycoprotein
|
|
gptkbp:target_audience
|
patients with Pompe disease
|
|
gptkbp:trade
|
gptkb:Myozyme
|
|
gptkbp:used_for
|
treatment of Pompe disease
|
|
gptkbp:bfsParent
|
gptkb:Genzyme
gptkb:Sanofi_Genzyme
gptkb:Genzyme_Corporation
|
|
gptkbp:bfsLayer
|
5
|