Matches in SemOpenAlex for { <https://semopenalex.org/work/W3148339990> ?p ?o ?g. }
Showing items 1 to 79 of
79
with 100 items per page.
- W3148339990 endingPage "S94" @default.
- W3148339990 startingPage "S93" @default.
- W3148339990 abstract "Clinical trials in DMD face challenges in selecting optimal endpoints and study cohorts to detect therapeutic efficacy. MR muscle biomarkers are objective and have high potential value for use in clinical trials offering excellent sensitivity to disease progression across a wide age range. A disease-drug trial model that describes the dynamics of MR measured fat fraction (FF) may help inform clinical trial design and identify early readouts that predict longer-term disease progression and response to treatment. A collaboration between industry, ImagingDMD and C-Path to accelerate drug development in DMD focuses on the development of statistical model-based clinical trial simulation (CTS) tool based on MR biomarker data acquired in the multicenter ImagingDMD study. The ImagingDMD study is a longitudinal natural history study in which functional outcome measures and MR biomarkers (FF and MRI-T2) have been collected from a large cohort of boys and young men with DMD annually for up to 10 years (>50% for more than 5 years). Non-linear mixed effects modelling was applied to describe the longitudinal increases of vastus lateralis (VL) muscle FF. The data set (N=122 subjects) was randomly divided into training (N=98) and test (N=24) datasets while maintaining balance in covariates. MR spectroscopy measures of VL FF significantly increased over time. The base model structure that best captured the course of the FF increase over time was a sigmoid Emax model. The model is clinically interpretable, with parameters describing maximum increase in VL FF score and the age at which VL FF is 50% of its eventual maximum. The longitudinal trajectory of VL FF in DMD can be captured by a statistical model, providing the opportunity to evaluate the effect of therapeutic interventions against the model-predicted disease trajectory and possibly broaden inclusion criteria for clinical trials in DMD. A CTS tool based on MR biomarkers has the potential to transform therapy development in DMD." @default.
- W3148339990 created "2021-04-13" @default.
- W3148339990 creator A5000311910 @default.
- W3148339990 creator A5003937899 @default.
- W3148339990 creator A5008558912 @default.
- W3148339990 creator A5017233978 @default.
- W3148339990 creator A5022894287 @default.
- W3148339990 creator A5023983627 @default.
- W3148339990 creator A5031529176 @default.
- W3148339990 creator A5048651139 @default.
- W3148339990 creator A5062897128 @default.
- W3148339990 creator A5070420986 @default.
- W3148339990 creator A5084106931 @default.
- W3148339990 creator A5084995630 @default.
- W3148339990 creator A5087832061 @default.
- W3148339990 date "2020-10-01" @default.
- W3148339990 modified "2023-10-14" @default.
- W3148339990 title "MUSCLE IMAGING – MRI" @default.
- W3148339990 doi "https://doi.org/10.1016/j.nmd.2020.08.163" @default.
- W3148339990 hasPublicationYear "2020" @default.
- W3148339990 type Work @default.
- W3148339990 sameAs 3148339990 @default.
- W3148339990 citedByCount "0" @default.
- W3148339990 crossrefType "journal-article" @default.
- W3148339990 hasAuthorship W3148339990A5000311910 @default.
- W3148339990 hasAuthorship W3148339990A5003937899 @default.
- W3148339990 hasAuthorship W3148339990A5008558912 @default.
- W3148339990 hasAuthorship W3148339990A5017233978 @default.
- W3148339990 hasAuthorship W3148339990A5022894287 @default.
- W3148339990 hasAuthorship W3148339990A5023983627 @default.
- W3148339990 hasAuthorship W3148339990A5031529176 @default.
- W3148339990 hasAuthorship W3148339990A5048651139 @default.
- W3148339990 hasAuthorship W3148339990A5062897128 @default.
- W3148339990 hasAuthorship W3148339990A5070420986 @default.
- W3148339990 hasAuthorship W3148339990A5084106931 @default.
- W3148339990 hasAuthorship W3148339990A5084995630 @default.
- W3148339990 hasAuthorship W3148339990A5087832061 @default.
- W3148339990 hasConcept C126322002 @default.
- W3148339990 hasConcept C185592680 @default.
- W3148339990 hasConcept C1862650 @default.
- W3148339990 hasConcept C2778279407 @default.
- W3148339990 hasConcept C2779134260 @default.
- W3148339990 hasConcept C2781197716 @default.
- W3148339990 hasConcept C535046627 @default.
- W3148339990 hasConcept C55493867 @default.
- W3148339990 hasConcept C71924100 @default.
- W3148339990 hasConcept C72563966 @default.
- W3148339990 hasConcept C99508421 @default.
- W3148339990 hasConceptScore W3148339990C126322002 @default.
- W3148339990 hasConceptScore W3148339990C185592680 @default.
- W3148339990 hasConceptScore W3148339990C1862650 @default.
- W3148339990 hasConceptScore W3148339990C2778279407 @default.
- W3148339990 hasConceptScore W3148339990C2779134260 @default.
- W3148339990 hasConceptScore W3148339990C2781197716 @default.
- W3148339990 hasConceptScore W3148339990C535046627 @default.
- W3148339990 hasConceptScore W3148339990C55493867 @default.
- W3148339990 hasConceptScore W3148339990C71924100 @default.
- W3148339990 hasConceptScore W3148339990C72563966 @default.
- W3148339990 hasConceptScore W3148339990C99508421 @default.
- W3148339990 hasLocation W31483399901 @default.
- W3148339990 hasOpenAccess W3148339990 @default.
- W3148339990 hasPrimaryLocation W31483399901 @default.
- W3148339990 hasRelatedWork W167982285 @default.
- W3148339990 hasRelatedWork W2071535500 @default.
- W3148339990 hasRelatedWork W2078446165 @default.
- W3148339990 hasRelatedWork W2155159475 @default.
- W3148339990 hasRelatedWork W2315085516 @default.
- W3148339990 hasRelatedWork W2603773853 @default.
- W3148339990 hasRelatedWork W2790789677 @default.
- W3148339990 hasRelatedWork W2975260264 @default.
- W3148339990 hasRelatedWork W3080646999 @default.
- W3148339990 hasRelatedWork W4210544780 @default.
- W3148339990 hasVolume "30" @default.
- W3148339990 isParatext "false" @default.
- W3148339990 isRetracted "false" @default.
- W3148339990 magId "3148339990" @default.
- W3148339990 workType "article" @default.