Cambridge -- U.S. scientists say they are starting a trial of a medication designed to treat the neurochemical defect underlying Fragile X syndrome.
Researchers at Seaside Therapeutics in Cambridge, Mass., said Fragile X syndrome -- the most common inherited cause of intellectual disability -- causes a range of developmental problems, including learning disabilities, cognitive impairment, attention deficit hyperactivity disorder and autism or autistic-like behavior.
People with Fragile X have DNA mutations in the FMR1 gene that, in effect, turn off the gene and prevent normal brain synaptic synthesis.
The new trial tests a Seaside Therapeutics' compound, STX107, which selectively and potently targets the synaptic defect.
