Fragile X syndrome (Autism), the most common cause of inherited intellectual impairment, may be treatable with a new medication called Arbaclofen, according to a study published in Science Translational Medicine.
Those affected by the syndrome display social avoidance and repetitive and sometimes violent behavior. Arbaclofen reduces GABA, a brain chemical which regulates the excitatory system in Fragile X patients, thereby reducing such symptoms.
"It's not a cure-all," said lead author Dr. Elizabeth Berry-Kravis of Rush University Medical Center in Chicago, the lead author of the study.
"But this is first example of a moderately large clinical trial that took a drug that was developed on the basis of research in mice and fly models of Fragile X, and theoretically corrects the deficit at the neuronal level," she added.
Arbaclofen is a derivative of baclofen (approved by the FDA), which treats muscle spasticity in conditions such as cerebal palsy.
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