Matches in SemOpenAlex for { <https://semopenalex.org/work/W2775386989> ?p ?o ?g. }
Showing items 1 to 83 of
83
with 100 items per page.
- W2775386989 endingPage "6" @default.
- W2775386989 startingPage "1" @default.
- W2775386989 abstract "Background Duchenne Muscular Dystrophy is a paediatric disorder resulting from a defective dystrophin gene. It causes progressive loss of muscle fibres, muscle weakness, and eventually loss of ambulation during adolescence, with death due to respiratory or cardiovascular complications soon afterwards. The drug eteplirsen has received support from medical experts and parents of affected children, but the FDA has delayed their decision for approval of this drug. Objective This study analysed the results of previous studies to assess the safety and efficacy of the eteplirsen, and is the first pooled-analysis of its kind. Methods A literature search of electronic databases was performed. Only human studies using eteplirsen were eligible. Results A total of four relevant clinical studies were identified. A pooled-analysis was performed using data relating to percentage dystrophin-positive fibres obtained from muscle biopsy, and the six-minute walk test (6 MWT). The average increase in percentage dystrophin-positive fibres after treatment with eteplirsen was 24.23% (range −4 to 78; SD 24.44%). The average rate of decline in distance walked was 65 metres (range −335 to 83; SD 100.08 m). Conclusions Whether or not this increase in percentage dystrophin-positive fibres and distance walked is clinically significant is unclear, and there is therefore a need for more clinical trials." @default.
- W2775386989 created "2017-12-22" @default.
- W2775386989 creator A5030410536 @default.
- W2775386989 creator A5066552691 @default.
- W2775386989 date "2018-03-01" @default.
- W2775386989 modified "2023-09-30" @default.
- W2775386989 title "Eteplirsen for paediatric patients with Duchenne muscular dystrophy: A pooled-analysis" @default.
- W2775386989 cites W1165233182 @default.
- W2775386989 cites W1524530894 @default.
- W2775386989 cites W1533447420 @default.
- W2775386989 cites W1913640993 @default.
- W2775386989 cites W1989566133 @default.
- W2775386989 cites W2023045813 @default.
- W2775386989 cites W2054680833 @default.
- W2775386989 cites W2067671642 @default.
- W2775386989 cites W2126068134 @default.
- W2775386989 cites W2138962751 @default.
- W2775386989 cites W2149543855 @default.
- W2775386989 cites W2157085976 @default.
- W2775386989 cites W2158156749 @default.
- W2775386989 cites W2161443721 @default.
- W2775386989 cites W2234725241 @default.
- W2775386989 cites W2327656791 @default.
- W2775386989 cites W2409170855 @default.
- W2775386989 cites W76368367 @default.
- W2775386989 doi "https://doi.org/10.1016/j.jocn.2017.10.082" @default.
- W2775386989 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/29254734" @default.
- W2775386989 hasPublicationYear "2018" @default.
- W2775386989 type Work @default.
- W2775386989 sameAs 2775386989 @default.
- W2775386989 citedByCount "23" @default.
- W2775386989 countsByYear W27753869892018 @default.
- W2775386989 countsByYear W27753869892019 @default.
- W2775386989 countsByYear W27753869892020 @default.
- W2775386989 countsByYear W27753869892021 @default.
- W2775386989 countsByYear W27753869892022 @default.
- W2775386989 countsByYear W27753869892023 @default.
- W2775386989 crossrefType "journal-article" @default.
- W2775386989 hasAuthorship W2775386989A5030410536 @default.
- W2775386989 hasAuthorship W2775386989A5066552691 @default.
- W2775386989 hasConcept C126322002 @default.
- W2775386989 hasConcept C141071460 @default.
- W2775386989 hasConcept C187212893 @default.
- W2775386989 hasConcept C2775934546 @default.
- W2775386989 hasConcept C2778943923 @default.
- W2775386989 hasConcept C2779030066 @default.
- W2775386989 hasConcept C2779999465 @default.
- W2775386989 hasConcept C2780247198 @default.
- W2775386989 hasConcept C2780394045 @default.
- W2775386989 hasConcept C535046627 @default.
- W2775386989 hasConcept C71924100 @default.
- W2775386989 hasConceptScore W2775386989C126322002 @default.
- W2775386989 hasConceptScore W2775386989C141071460 @default.
- W2775386989 hasConceptScore W2775386989C187212893 @default.
- W2775386989 hasConceptScore W2775386989C2775934546 @default.
- W2775386989 hasConceptScore W2775386989C2778943923 @default.
- W2775386989 hasConceptScore W2775386989C2779030066 @default.
- W2775386989 hasConceptScore W2775386989C2779999465 @default.
- W2775386989 hasConceptScore W2775386989C2780247198 @default.
- W2775386989 hasConceptScore W2775386989C2780394045 @default.
- W2775386989 hasConceptScore W2775386989C535046627 @default.
- W2775386989 hasConceptScore W2775386989C71924100 @default.
- W2775386989 hasLocation W27753869891 @default.
- W2775386989 hasLocation W27753869892 @default.
- W2775386989 hasOpenAccess W2775386989 @default.
- W2775386989 hasPrimaryLocation W27753869891 @default.
- W2775386989 hasRelatedWork W1970270461 @default.
- W2775386989 hasRelatedWork W1972266802 @default.
- W2775386989 hasRelatedWork W1983715694 @default.
- W2775386989 hasRelatedWork W1996504692 @default.
- W2775386989 hasRelatedWork W2060554805 @default.
- W2775386989 hasRelatedWork W2108474119 @default.
- W2775386989 hasRelatedWork W2111939281 @default.
- W2775386989 hasRelatedWork W2154747396 @default.
- W2775386989 hasRelatedWork W2299139921 @default.
- W2775386989 hasRelatedWork W2322780762 @default.
- W2775386989 hasVolume "49" @default.
- W2775386989 isParatext "false" @default.
- W2775386989 isRetracted "false" @default.
- W2775386989 magId "2775386989" @default.
- W2775386989 workType "article" @default.