Properties (59)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:activeDuring |
AAV1-LPLS447X
|
| gptkbp:administrativeDivision |
single dose
hospital or clinic directly into bloodstream |
| gptkbp:approves |
gptkb:European_Union
|
| gptkbp:clinicalTrials |
Europe
completed Phase III published in medical journals positive results developed for use genetic testing required over 50 patients support research and awareness uniQure |
| gptkbp:community_service |
available through manufacturer
|
| gptkbp:customerFeedback |
generally positive
|
| gptkbp:customizationOptions |
enzyme replacement therapy
|
| gptkbp:deployedTo |
2017
|
| gptkbp:developedBy |
uniQure
|
| gptkbp:diseaseResistance |
LPL_deficiency
|
| gptkbp:geneticDiversity |
gptkb:LPL_gene
recommended for patients |
| gptkbp:hasPopulation |
adults and children
|
| gptkbp:healthcare |
required for administration
high due to complexity required after treatment confirmed_LPL_deficiency |
| gptkbp:historicalResearch |
focused on broader applications.
|
| https://www.w3.org/2000/01/rdf-schema#label |
Glybera
|
| gptkbp:impact |
one-time treatment
improved lipid metabolism lipid levels checked regularly long-term effect |
| gptkbp:insuranceAccepted |
limited availability
|
| gptkbp:lastProduced |
2012
|
| gptkbp:lengthOverall |
ongoing studies
|
| gptkbp:mandates |
treatment_of_LPL_deficiency
|
| gptkbp:market |
2012
limited patient population |
| gptkbp:marketedAs |
LPLD
|
| gptkbp:marketSegment |
not applicable
|
| gptkbp:offers |
over $1 million
|
| gptkbp:producedBy |
gptkb:uniQure_Biopharma_N.V.
|
| gptkbp:regulatoryCompliance |
gptkb:European_Medicines_Agency
withdrawn from market |
| gptkbp:relatedPatent |
other gene therapies
|
| gptkbp:research |
private investment
|
| gptkbp:research_areas |
rare diseases
|
| gptkbp:researchField |
genetic disorders
|
| gptkbp:researchInterest |
with academic institutions
multiple peer-reviewed articles |
| gptkbp:route |
intravenous injection
|
| gptkbp:safetyFeatures |
generally well tolerated
|
| gptkbp:sideEffect |
injection site reactions
|
| gptkbp:succeededBy |
variable
|
| gptkbp:targets |
lipoprotein lipase deficiency
|
| gptkbp:triggerType |
gene replacement therapy
|