title: Assessing cognitive improvement in people with Down syndrome: important considerations for drug-efficacy trials.
abstract: Experimental research over just the past decade has raised the possibility that learning deficits connected to Down syndrome (DS) might be effectively managed by medication. In the current chapter, we touch on some of the work that paved the way for these advances and discuss the challenges associated with translating them. In particular, we highlight sources of phenotypic variability in the DS population that are likely to impact performance assessments. Throughout, suggestions are made on how to detect meaningful changes in cognitive-adaptive function in people with DS during drug treatment. The importance of within-subjects evaluation is emphasized.
authors.affiliation: Department of Physiology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA, ffernan6@jhmi.edu.
authors.forename: Fabian
authors.initials: F
authors.lastname: Fernandez
authors.forename: Roger H
authors.initials: RH
authors.lastname: Reeves
mesh_terms: D000818:Animals; D001522:Behavior, Animal / Q000187:drug effects; D001921:Brain / Q000187:drug effects* / Q000503:physiopathology; D002986:Clinical Trials as Topic / Q000379:methods*; D003071:Cognition / Q000187:drug effects*; D004195:Disease Models, Animal; D004314:Down Syndrome / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology / Q000523:psychology; D020022:Genetic Predisposition to Disease; D006801:Humans; D007858:Learning / Q000187:drug effects; D018697:Nootropic Agents / Q000627:therapeutic use*; D010641:Phenotype; D020127:Recovery of Function; D012107:Research Design; D016896:Treatment Outcome
chemical_list: D018697:Nootropic Agents