Comparing ability and norm-referenced scores as clinical trial outcomes for neurodevelopmental disabilities: a simulation study

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SUMMARY

    The recent momentum in GCAND pharmaceutical clinical trials (e_g, Fragile X syndrome;) and gene or genomic therapy (e_g, Angelman syndrome and Rett syndrome ) has spurred interest in the psychometric profiles of measures intended for developmental concepts. Norm-referenced scores (which may be referred to as "standard" or "scaled" scores) are distribution-based derivations, meaning that they are an empirically based estimate of the proportion of the proband`s peer population expected to achieve the same score or lower. Norm-referenced scores are also known to exhibit poor reliability at extreme values (e_g, ), where "extreme . . .

     

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