Clinical trials are set to begin on a drug which could improve cognitive functioning for individuals with fragile X syndrome, the leading cause of intellectual disability, researchers say.

The drug, currently called STX107, targets altered brain connections that appear in individuals with fragile X. Researchers hope that the drug will force the brain to make normal connections therefore improving learning capabilities in those with the disorder.

Researchers are beginning initial tests of the drug on healthy adults to determine if it is safe in humans. If successful, they will try the drug in adults with fragile X and later on in children.

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“Initiation of the STX107 safety study is a momentous first step in the development of a potentially disease-modifying treatment for brain development disorders, such as fragile X Syndrome and autism,” according to Randall Carpenter, president and chief executive officer of Seaside Therapeutics, which is conducting the trials with funding from the National Institutes of Health. “We believe that normalizing signaling in the mGluR5 pathway with STX107 provides a compelling opportunity to correct or improve the course of fragile X syndrome.”

Fragile X is an inherited disorder and is the most common cause of intellectual disability. It is found in 1 in 4,000 boys and 1 in 6,000 girls.

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