Matches in SemOpenAlex for { <https://semopenalex.org/work/W1831130622> ?p ?o ?g. }
- W1831130622 endingPage "526" @default.
- W1831130622 startingPage "513" @default.
- W1831130622 abstract "N-Butyldeoxynojirimycin (NB-DNJ, miglustat 'Zavesca') is an orally active iminosugar which inhibits the biosynthesis of macromolecular substrates that accumulate pathologically in glycosphingolipidoses. Clinical trials of NB-DNJ in patients with Gaucher's disease demonstrate the therapeutic potential of such substrate inhibitors in the glycolipid storage disorders. However, macrophage-targetted enzyme replacement using intravenous mannose-terminated human glucocerebrosidase (imiglucerase, Cerezyme) is highly effective in ameliorating many of the manifestations of Gaucher's disease and is a treatment in widespread use. Given that imiglucerase and miglustat are now both licensed for the treatment of Gaucher's disease, there is a need to review their therapeutic status. Here the treatment of type 1 (non-neuronopathic) Gaucher disease is evaluated with particular reference to the emerging role of oral N-butyldeoxynojirimycin (miglustat) as a substrate-reducing agent. This position statement represents the consensus viewpoint of an independent international advisory council to the European Working Group on Gaucher Disease." @default.
- W1831130622 created "2016-06-24" @default.
- W1831130622 creator A5009904801 @default.
- W1831130622 creator A5013935159 @default.
- W1831130622 creator A5014671077 @default.
- W1831130622 creator A5017093187 @default.
- W1831130622 creator A5022060295 @default.
- W1831130622 creator A5024669037 @default.
- W1831130622 creator A5024894157 @default.
- W1831130622 creator A5032207811 @default.
- W1831130622 creator A5035270139 @default.
- W1831130622 creator A5037829084 @default.
- W1831130622 creator A5039674178 @default.
- W1831130622 creator A5042102713 @default.
- W1831130622 creator A5046731360 @default.
- W1831130622 creator A5049636476 @default.
- W1831130622 creator A5050458862 @default.
- W1831130622 creator A5069257242 @default.
- W1831130622 creator A5080995646 @default.
- W1831130622 creator A5084371637 @default.
- W1831130622 creator A5091262663 @default.
- W1831130622 creator A5059926348 @default.
- W1831130622 date "2003-09-01" @default.
- W1831130622 modified "2023-10-12" @default.
- W1831130622 title "The role of the iminosugar <i>N</i>‐butyldeoxynojirimycin (miglustat) in the management of type I (non‐neuronopathic) Gaucher disease: A position statement" @default.
- W1831130622 cites W129763758 @default.
- W1831130622 cites W1485152739 @default.
- W1831130622 cites W1542335975 @default.
- W1831130622 cites W1592147357 @default.
- W1831130622 cites W1966296534 @default.
- W1831130622 cites W1968758382 @default.
- W1831130622 cites W1969551374 @default.
- W1831130622 cites W1978453396 @default.
- W1831130622 cites W1981340489 @default.
- W1831130622 cites W1981725999 @default.
- W1831130622 cites W1983064528 @default.
- W1831130622 cites W1992088688 @default.
- W1831130622 cites W1995165467 @default.
- W1831130622 cites W1998164429 @default.
- W1831130622 cites W2000125278 @default.
- W1831130622 cites W2017680639 @default.
- W1831130622 cites W2030498863 @default.
- W1831130622 cites W2033060552 @default.
- W1831130622 cites W2043412667 @default.
- W1831130622 cites W2051654324 @default.
- W1831130622 cites W2053490488 @default.
- W1831130622 cites W2059273086 @default.
- W1831130622 cites W2064324964 @default.
- W1831130622 cites W2076780197 @default.
- W1831130622 cites W2077868231 @default.
- W1831130622 cites W2086382529 @default.
- W1831130622 cites W2086672639 @default.
- W1831130622 cites W2093722439 @default.
- W1831130622 cites W2119593625 @default.
- W1831130622 cites W2124663723 @default.
- W1831130622 cites W2126057638 @default.
- W1831130622 cites W2126583938 @default.
- W1831130622 cites W2132911597 @default.
- W1831130622 cites W2135424735 @default.
- W1831130622 cites W2140474525 @default.
- W1831130622 cites W2143447170 @default.
- W1831130622 cites W2233399397 @default.
- W1831130622 cites W230215977 @default.
- W1831130622 cites W2322128806 @default.
- W1831130622 cites W2371946969 @default.
- W1831130622 cites W2897831061 @default.
- W1831130622 cites W4256541688 @default.
- W1831130622 cites W4296588185 @default.
- W1831130622 cites W82926723 @default.
- W1831130622 doi "https://doi.org/10.1023/a:1025902113005" @default.
- W1831130622 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/14605497" @default.
- W1831130622 hasPublicationYear "2003" @default.
- W1831130622 type Work @default.
- W1831130622 sameAs 1831130622 @default.
- W1831130622 citedByCount "222" @default.
- W1831130622 countsByYear W18311306222012 @default.
- W1831130622 countsByYear W18311306222013 @default.
- W1831130622 countsByYear W18311306222014 @default.
- W1831130622 countsByYear W18311306222015 @default.
- W1831130622 countsByYear W18311306222016 @default.
- W1831130622 countsByYear W18311306222017 @default.
- W1831130622 countsByYear W18311306222018 @default.
- W1831130622 countsByYear W18311306222019 @default.
- W1831130622 countsByYear W18311306222020 @default.
- W1831130622 countsByYear W18311306222021 @default.
- W1831130622 countsByYear W18311306222022 @default.
- W1831130622 countsByYear W18311306222023 @default.
- W1831130622 crossrefType "journal-article" @default.
- W1831130622 hasAuthorship W1831130622A5009904801 @default.
- W1831130622 hasAuthorship W1831130622A5013935159 @default.
- W1831130622 hasAuthorship W1831130622A5014671077 @default.
- W1831130622 hasAuthorship W1831130622A5017093187 @default.
- W1831130622 hasAuthorship W1831130622A5022060295 @default.
- W1831130622 hasAuthorship W1831130622A5024669037 @default.
- W1831130622 hasAuthorship W1831130622A5024894157 @default.
- W1831130622 hasAuthorship W1831130622A5032207811 @default.
- W1831130622 hasAuthorship W1831130622A5035270139 @default.
- W1831130622 hasAuthorship W1831130622A5037829084 @default.