New drug target identified for Fragile X syndrome

New drug target identified for Fragile X syndrome

UCLA Health researchers have identified a potential drug target for treating Fragile X syndrome, the most common genetic cause of intellectual disability and autism that affects roughly one in 2,000 boys.

Drug Target for Fragile X Syndrome Identified Through Preclinical Study

UCLA Health researchers have identified a potential drug target for treating fragile X syndrome (FXS), the most common genetic cause of intellectual disability and autism that affects roughly one in 2,000 boys. Fragile X syndrome is caused by a mutation in a single gene, FMR1, that results in the loss of a protein critical for normal brain development and function. Headed by Carlos Portera-Cailliau, MD, PhD, professor of neurology at UCLA and me…

Novel Drug Target to Reverse Fragile X Syndrome Found

Can an overactive synaptic protein hold the key to treating the most common genetic cause of autism? A new study identifies the protein EPAC2 as a potent drug target for reversing the behavioral and neurological deficits of Fragile X syndrome.