Statements (63)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gptkb:physicist
|
| gptkbp:activeDuring |
elosulfase alfa
|
| gptkbp:administrativeDivision |
4 hours
|
| gptkbp:cancellationReason |
based on clinical response
|
| gptkbp:clinicalTrials |
gptkb:BioMarin_Pharmaceutical
over 200 weekly multiple countries improved quality of life Phase III long-term treatment in peer-reviewed journals included in treatment protocols available for financial assistance improved walking ability not a cure reduced urinary keratan sulfate |
| gptkbp:community_service |
available
|
| gptkbp:contraindication |
fatigue
headache nausea fever vomiting rash hypersensitivity to elosulfase alfa |
| gptkbp:customerFeedback |
generally positive
|
| gptkbp:date |
2014-02-14
|
| gptkbp:discontinued |
Yes
|
| gptkbp:dosageForm |
solution for infusion
|
| gptkbp:drugInterdiction |
none significant reported
increases enzyme activity steady-state concentration achieved after multiple doses |
| gptkbp:economicImpact |
improves patient outcomes
|
| gptkbp:formulation |
sterile solution
|
| gptkbp:hasPopulation |
children and adults
|
| gptkbp:healthcare |
recommended
required during treatment important for adherence diagnosed_with_Morquio_A |
| https://www.w3.org/2000/01/rdf-schema#label |
Vimizim
|
| gptkbp:impact |
reduce symptoms
|
| gptkbp:is_used_in |
not fully known
|
| gptkbp:lastProduced |
gptkb:United_States
|
| gptkbp:mandates |
Mucopolysaccharidosis_type_IVA
|
| gptkbp:manufacturer |
gptkb:BioMarin_Pharmaceutical
|
| gptkbp:market |
global
|
| gptkbp:marketedAs |
gptkb:Vimizim
|
| gptkbp:marketSegment |
ongoing
|
| gptkbp:offers |
high
|
| gptkbp:packaging |
single-use vial
|
| gptkbp:regulatoryCompliance |
FDA_approved
|
| gptkbp:relatedPatent |
Mucopolysaccharidosis
|
| gptkbp:research |
provided_by_BioMarin
|
| gptkbp:researchFocus |
rare diseases
|
| gptkbp:route |
intravenous infusion
|
| gptkbp:safetyFeatures |
generally well tolerated
recommended during treatment |
| gptkbp:sideEffect |
infusion-related reactions
|
| gptkbp:storage |
refrigerated
|
| gptkbp:supplyChain |
cold chain logistics
|
| gptkbp:triggerType |
replaces deficient enzyme
|
| gptkbp:typeOfInsurance |
varies by plan
|
| gptkbp:usedFor |
treatment_of_Morquio_A_syndrome
|