Abstract Objective Fragile X syndrome (FXS) has been the neurodevelopmental disorder
with the most active translation of preclinical breakthroughs into clinical trials. This process …

Rett syndrome is a rare, genetic neurodevelopmental disorder. Trofinetide is a synthetic
analog of glycine–proline–glutamate, the N-terminal tripeptide of the insulin-like growth …

Down syndrome (DS), also known as trisomy 21, is the most common genetic cause of
intellectual disability (ID). Although ID can be mild, the average intelligence quotient is in the …

Aberrant alternative splicing of mRNAs results in dysregulated gene expression in multiple
neurological disorders. Here, we show that hundreds of mRNAs are incorrectly expressed …

Introduction Rett syndrome (RTT) is a debilitating neurodevelopmental disorder with no
approved treatments. Trofinetide is a synthetic analog of glycine–proline–glutamate, the N …

Increasingly individuals with intellectual and developmental disabilities, including Down
syndrome, are being targeted for clinical trials. However, a challenge exists in effectively …

Background The lack of robust and reliable clinical biomarkers in Fragile X Syndrome (FXS),
the most common inherited form of intellectual disability, has limited the successful …

Preclinical data suggest that inhibition of the metabotropic glutamate receptor 5 (mGluR5)
receptor might hold therapeutic benefits in Fragile X syndrome (FXS). Treatment of Fmr1 …

Advances in genomicshaveusheredinpromisingthera-pies tailored to the individual.
Personalized medicine is promoted and has begun to positively influence care. For …

Despite the significance of efforts to understand the biological basis of autism, progress in
this area has been hindered, in part, by the considerable heterogeneity in the disorder …