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- W4286543356 abstract "Development in domains like motor ability, thinking and learning, social abilities, and communication are important areas of concern for individuals affected by genetic conditions associated with neurodevelopmental disorder (GCAND). Challenges associated with clinical trials in rare conditions, such as limited information about natural history, small sample size, and heterogeneous presentation, have been well-described. However, little attention has been paid to the additional complications conferred by use of developmental outcomes in these studies. Change is inherent to developmental concepts, which complicates the establishment of psychometric profiles that is already challenged by heterogeneous populations. Further, change in developmental concepts during a clinical trial may be difficult to interpret. This is especially true for disorders which are associated with significant delay or even decline, where decreases in age-based normative scores may be caused by active degeneration or slower-than-expected growth. Choices in the selection of the concept of interest, the clinical outcome assessment, and the endpoint may mitigate these issues. In this chapter, we review essential background information about those choices and discuss additional considerations and unresolved challenges that investigators in GCAND may face when the concept is developmental in nature, in order to prompt future efforts." @default.
- W4286543356 created "2022-07-22" @default.
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- W4286543356 date "2022-01-01" @default.
- W4286543356 modified "2023-10-18" @default.
- W4286543356 title "Psychometric perspectives on developmental outcome and endpoint selection in treatment trials for genetic conditions associated with neurodevelopmental disorder" @default.
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- W4286543356 doi "https://doi.org/10.1016/bs.irrdd.2022.05.001" @default.
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