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Medical News & Perspectives
January 26, 2011

Scientists Find Promising Therapies for Fragile X and Down Syndromes

JAMA. 2011;305(4):344-346. doi:10.1001/jama.2010.1960

Clinicians have long viewed the intellectual disabilities associated with developmental disorders, such as fragile X and Down syndromes, as irreversible or untreatable. However, emerging data suggest that a number of potential drug therapies targeting the molecular pathways involved in these disorders may one day help to improve cognition, memory, and behavior in patients with these genetic conditions.

Advances in the understanding of the genetic and molecular basis of developmental disorders, as well as the creation of mouse models for these conditions, have led scientists studying them to some surprising insights. Even later in life, mice with Down syndrome or fragile X syndrome (FXS) that are given targeted treatment can experience improvements in cognitive function. Findings from such animal studies have paved the way to human trials to assess some of these potential therapies, with some experimental therapies for FXS already entering late-stage clinical trials.

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