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|Titolo:||204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The Netherlands.|
|Autori interni:||FERLINI, Alessandra|
|Data di pubblicazione:||2014|
|Abstract:||The workshop intended to increase cooperative and harmonized effort in designing common research plans for biomarker discovery and development, and bridging clinical measures and biomarker research. A comprehensive overview on biomarker research in DMD was presented, highlighting some established and yet unpublished data, and encouraging data sharing and collaboration, which is necessary for validation of biomarker data in larger cohorts. A highly multidisciplinary group attended the workshop. The focus of the workshop was on Duchenne muscular dystrophy including ongoing trials, extremely well defined pathomechanisms, currently available exploratory biomarkers as a prototype forthis field. Nevertheless the output of this workshop is expected to be useful for other NMDs. This collaborative EU and USA workshop aimed at establishing a large cooperation in order to increase critical mass, patient data and bio-samples to allow robust biomarker validation. The following sessions were focused on: 1) the need for additional biomarkers for DMD, 2) strategies to identify and employ biomarkers with focus on novel high throughput technologies, 3) known biomarkers, 4) outcome measures in DMD and how biomarkers may be surrogates for outcome measures, 5) role of modifying factors in DMD, and 6) planning the future for biomarker identification. This last session was organized in concurrent roundtables in order to concretize the output of the workshop in future actions. The key topics considered to set up collaborations were registries and data sharing, biomarkers in clinical trials and integration with the clinical outcome measures and EMA requirement for biomarker regulation.|
|Appare nelle tipologie:||03.1 Articolo su rivista|
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