A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease

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Annotation Summary

Term Occurence Count Dictionary
miglustat 2 endocrinologydiseasesdrugs
Fabry disease 1 endocrinologydiseases
eliglustat 4 endocrinologydiseasesdrugs
lysosomal storage disease 2 endocrinologydiseases

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Select Drug Character Offset Drug Term Instance
eliglustat 4153 [[8]], velaglucerase alfa [[9]] and taliglucerase alfa [[10]]) and substrate reduction therapy (SRT) ( eliglustat [[11], [12]] and miglustat [[13]]). Both ERTs and SRTs require lifelong treatment. ERTs are administered
eliglustat 4344 treatment. ERTs are administered intravenously, usually every two weeks in an outpatient procedure. The SRT eliglustat was recently approved by both the US Food and Drug Administration (FDA) in 2014 [[14]] and the European
eliglustat 4933 administered orally on daily basis.Intravenously-administered ERT imiglucerase and orally-administered SRT eliglustat have demonstrated comparable efficacy [[11]], however, the value of oral versus intravenous treatment
eliglustat 6765 literature describing available therapies and of the corresponding product labels (i.e. imiglucerase and eliglustat ) was undertaken. In total, five health states were developed. First, a benchmark state was described
miglustat 4181 [[9]] and taliglucerase alfa [[10]]) and substrate reduction therapy (SRT) (eliglustat [[11], [12]] and miglustat [[13]]). Both ERTs and SRTs require lifelong treatment. ERTs are administered intravenously, usually
miglustat 4675 with GD1 who have compatible CYP2D6 metaboliser phenotypes (> 90% of patients) [[16], [17]]. The SRT miglustat is also approved by FDA and EMA as a second-line treatment for adults with GD1 who cannot be treated
Select Disease Character Offset Disease Term Instance
Fabry disease 22062 experiment study exploring the value that people placed on the different features of treatments for Fabry disease showed that oral treatment was preferred significantly over intravenous treatment [[38]].There are several
lysosomal storage disease 2923 substrate reduction therapy. Gaucher disease affects patients of all ages and is one of the most common lysosomal storage disease s with a worldwide prevalence of approximately 1/100,000 and approximately 1/855 in the Ashkenazi Jewish
lysosomal storage disease 21817 difference in HRQL impact for patients.Our findings in Gaucher disease could also be relevant to other lysosomal storage disease s linked to enzyme deficiency and for which oral and intravenous form of therapy is available. A recent

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