Down syndrome (DS) is a genetic disorder that results from the triplication
of chromosome 21. Accompanying this disorder are a number of medical conditions
including intellectual disability and early- onset Alzheimer’s disease (AD). Our
understanding of the molecular basis for these has expanded substantially in the past
decade, both in patients and relevant animal models. In particular, exciting advances in
therapies that modulate neurotransmitters to improve cognitive function have reached
clinical trials. Moreover, increased insight into the pathogenesis of AD in DS has
motivated clinical trials for AD in DS. Below, therapeutic strategies that have been
deployed in clinical trials in DS will be discussed as well as their underlying scientific
rational.
Keywords: Alzheimer’s disease, clinical trials, pharmacotherapy.
