Updated report on tools to measure outcomes of clinical trials in fragile X syndrome
Updated report on tools to measure outcomes of clinical trials in fragile X syndrome
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England: BioMed Central Ltd
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English
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England: BioMed Central Ltd
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Fragile X syndrome (FXS) has been the neurodevelopmental disorder with the most active translation of preclinical breakthroughs into clinical trials. This process has led to a critical assessment of outcome measures, which resulted in a comprehensive review published in 2013. Nevertheless, the disappointing outcome of several recent phase III drug trials in FXS, and parallel efforts at evaluating behavioral endpoints for trials in autism spectrum disorder (ASD), has emphasized the need for re-assessing outcome measures and revising recommendations for FXS.
After performing an extensive database search (PubMed, Food and Drug Administration (FDA)/National Institutes of Health (NIH)'s www.ClinicalTrials.gov, etc.) to determine progress since 2013, members of the Working Groups who published the 2013 Report evaluated the available outcome measures for FXS and related neurodevelopmental disorders using the COSMIN grading system of levels of evidence. The latter has also been applied to a British survey of endpoints for ASD. In addition, we also gen...
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Updated report on tools to measure outcomes of clinical trials in fragile X syndrome
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TN_cdi_doaj_primary_oai_doaj_org_article_0fef9f0727e54e7ca2fc003359a535e7
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_0fef9f0727e54e7ca2fc003359a535e7
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ISSN
1866-1947
E-ISSN
1866-1955
DOI
10.1186/s11689-017-9193-x
