FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2170533045> ?p ?o ?g. }

• W2170533045 endingPage "966" @default.
• W2170533045 startingPage "961" @default.
• W2170533045 abstract "Gaucher disease is caused by an enzymatic defect with consequent accumulation of glucocerebroside. Type I, the non–neuronopathic form, is rather common and panethnic. Patients may present with hepatosplenomegaly, anaemia, thrombocytopenia and skeletal or lung involvement. Enzyme replacement therapy ameliorates disease symptoms and signs; however, it involves lifelong intravenous therapy, is costly and is incapable of crossing the blood–brain barrier. Substrate reduction with N –butyldeoxynojirimycin (OGT 918) is a harbinger of oral iminosugars for glycolipid storage disorders. Long–term data in the seminal trial (100 mg three times per day), demonstrate safety and efficacy in adult type I patients naive to enzyme therapy, and suggest its application in patients unwilling or unable to receive enzyme replacement and tolerating side effects, including diarrhoea, weight loss, tremor and peripheral neuropathy (mostly reversible with dose reduction or withdrawal). Dose dependency was demonstrated with 50 mg three times per day. In patients stabilized on enzyme therapy switched from or in combination with enzyme, no deterioration in disease parameters was seen but side effects were as above. Although efficacy is less dramatic than enzyme treatment, it may be that plateaux are achieved asymptotically so therapeutic outcomes with OGT 918 may ultimately be comparable. Yet, given the above side effects and the lack of long–term experience, patients with very mild manifestations would probably not be appropriate candidates." @default.
• W2170533045 created "2016-06-24" @default.
• W2170533045 creator A5009904801 @default.
• W2170533045 creator A5037829084 @default.
• W2170533045 date "2003-05-29" @default.
• W2170533045 modified "2023-10-10" @default.
• W2170533045 title "Gaucher disease and the clinical experience with substrate reduction therapy" @default.
• W2170533045 cites W1485152739 @default.
• W2170533045 cites W1966296534 @default.
• W2170533045 cites W1968758382 @default.
• W2170533045 cites W1976904815 @default.
• W2170533045 cites W1980496044 @default.
• W2170533045 cites W2028623514 @default.
• W2170533045 cites W2030498863 @default.
• W2170533045 cites W2032826794 @default.
• W2170533045 cites W2048088579 @default.
• W2170533045 cites W2062899231 @default.
• W2170533045 cites W2086382529 @default.
• W2170533045 cites W2095190172 @default.
• W2170533045 cites W2097972170 @default.
• W2170533045 cites W2102462689 @default.
• W2170533045 cites W2105362377 @default.
• W2170533045 cites W2132911597 @default.
• W2170533045 cites W2135424735 @default.
• W2170533045 cites W2143301220 @default.
• W2170533045 cites W2156493486 @default.
• W2170533045 cites W2168903900 @default.
• W2170533045 cites W2170919408 @default.
• W2170533045 cites W2322128806 @default.
• W2170533045 cites W4296588185 @default.
• W2170533045 doi "https://doi.org/10.1098/rstb.2003.1272" @default.
• W2170533045 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/1693183" @default.
• W2170533045 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/12803930" @default.
• W2170533045 hasPublicationYear "2003" @default.
• W2170533045 type Work @default.
• W2170533045 sameAs 2170533045 @default.
• W2170533045 citedByCount "69" @default.
• W2170533045 countsByYear W21705330452012 @default.
• W2170533045 countsByYear W21705330452013 @default.
• W2170533045 countsByYear W21705330452014 @default.
• W2170533045 countsByYear W21705330452015 @default.
• W2170533045 countsByYear W21705330452016 @default.
• W2170533045 countsByYear W21705330452018 @default.
• W2170533045 countsByYear W21705330452020 @default.
• W2170533045 countsByYear W21705330452022 @default.
• W2170533045 crossrefType "journal-article" @default.
• W2170533045 hasAuthorship W2170533045A5009904801 @default.
• W2170533045 hasAuthorship W2170533045A5037829084 @default.
• W2170533045 hasBestOaLocation W21705330452 @default.
• W2170533045 hasConcept C126322002 @default.
• W2170533045 hasConcept C181199279 @default.
• W2170533045 hasConcept C21850170 @default.
• W2170533045 hasConcept C2779134260 @default.
• W2170533045 hasConcept C2779440204 @default.
• W2170533045 hasConcept C2779969927 @default.
• W2170533045 hasConcept C2779994418 @default.
• W2170533045 hasConcept C2780674200 @default.
• W2170533045 hasConcept C511355011 @default.
• W2170533045 hasConcept C544821477 @default.
• W2170533045 hasConcept C55493867 @default.
• W2170533045 hasConcept C71924100 @default.
• W2170533045 hasConcept C86803240 @default.
• W2170533045 hasConcept C90924648 @default.
• W2170533045 hasConceptScore W2170533045C126322002 @default.
• W2170533045 hasConceptScore W2170533045C181199279 @default.
• W2170533045 hasConceptScore W2170533045C21850170 @default.
• W2170533045 hasConceptScore W2170533045C2779134260 @default.
• W2170533045 hasConceptScore W2170533045C2779440204 @default.
• W2170533045 hasConceptScore W2170533045C2779969927 @default.
• W2170533045 hasConceptScore W2170533045C2779994418 @default.
• W2170533045 hasConceptScore W2170533045C2780674200 @default.
• W2170533045 hasConceptScore W2170533045C511355011 @default.
• W2170533045 hasConceptScore W2170533045C544821477 @default.
• W2170533045 hasConceptScore W2170533045C55493867 @default.
• W2170533045 hasConceptScore W2170533045C71924100 @default.
• W2170533045 hasConceptScore W2170533045C86803240 @default.
• W2170533045 hasConceptScore W2170533045C90924648 @default.
• W2170533045 hasIssue "1433" @default.
• W2170533045 hasLocation W21705330451 @default.
• W2170533045 hasLocation W21705330452 @default.
• W2170533045 hasLocation W21705330453 @default.
• W2170533045 hasLocation W21705330454 @default.
• W2170533045 hasOpenAccess W2170533045 @default.
• W2170533045 hasPrimaryLocation W21705330451 @default.
• W2170533045 hasRelatedWork W1837812877 @default.
• W2170533045 hasRelatedWork W1982869943 @default.
• W2170533045 hasRelatedWork W2072241333 @default.
• W2170533045 hasRelatedWork W2118170260 @default.
• W2170533045 hasRelatedWork W2161617214 @default.
• W2170533045 hasRelatedWork W2281150386 @default.
• W2170533045 hasRelatedWork W2407717434 @default.
• W2170533045 hasRelatedWork W2567269117 @default.
• W2170533045 hasRelatedWork W2573994062 @default.
• W2170533045 hasRelatedWork W2616301251 @default.
• W2170533045 hasVolume "358" @default.
• W2170533045 isParatext "false" @default.
• W2170533045 isRetracted "false" @default.
• W2170533045 magId "2170533045" @default.

• next