Many available drugs have been repurposed as treatments for neurodevelopmental
disorders. In the specific case of fragile X syndrome, many clinical trials of available drugs …

Neurodevelopmental disorders such as fragile X syndrome (FXS) result in lifelong cognitive
and behavioural deficits and represent a major public health burden. FXS is the most …

Highlights•Fragile X pathophysiological mechanisms were identified in animal
models.•Targeted treatments are being developed based on molecular defects.•The first …

Ongoing treatments for genetic developmental disorders of the central nervous system are
mostly symptomatic and do not correct the genetic cause. Recent identification of common …

Intellectual disability (ID; mental retardation) is considered an immutable condition. Current
medical practices are aimed at relieving symptoms and not at altering the underlying …

In the last decade there has been a revolution in terms of genetic findings in
neurodevelopmental disorders (NDDs), with many discoveries critical for understanding …

Preclinical studies using animal models of fragile X syndrome have yielded several agents
that rescue a wide variety of phenotypes. However, translation of these treatments to …

Fragile X syndrome (FXS) is one of the most prevalent and well-studied monogenetic
causes of intellectual disability and autism and, although rare, its high penetrance makes it a …

Fragile X syndrome (FXS) is the leading known cause of inherited intellectual disability and
autism spectrum disorder. It is caused by a mutation of the fragile X mental retardation 1 …

Clinical trials for neurodevelopmental disorders: At a therapeutic frontier | Science Translational
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