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Drug Shows Promise Against Fragile X Syndrome, Possibly Autism
Date:9/19/2012

By Randy Dotinga
HealthDay Reporter

WEDNESDAY, Sept. 19 (HealthDay News) -- In a preliminary study, scientists are reporting that an experimental pill designed to treat fragile X syndrome -- a genetic condition tied to intellectual impairment, or "mental retardation," and some cases of autism -- may help patients develop better social skills.

The scientists are continuing to study the medication and have launched research into whether it may also help social withdrawal in those with autism. However, it's still not clear if the drug works, nor do its developers know how much it might cost. It's also not known whether other medications in development might do a better job, either in conjunction with the new drug or instead of it.

Still, the results so far are promising, and the medication "could be an important model for developing treatments for autism," said Dr. Michael Tranfaglia, medical director of the FRAXA Research Foundation, a nonprofit organization seeking better treatments for fragile X syndrome.

Fragile X syndrome is caused by the loss of the gene for fragile X mental retardation protein. The condition affects an estimated 100,000 people in the United States, potentially leading to mental retardation, epilepsy, autism and abnormal body growth. There is no cure for fragile X syndrome.

Essentially, the lack of the gene weakens the wiring of the brain, said Stephen Warren, chairman of human genetics at Emory University School of Medicine in Atlanta. People with the condition usually must live in group homes, although they're often able to work, he added.

Several studies have been launched into the drug -- known as arbaclofen -- as a treatment for fragile X syndrome and related autism-type conditions. The new study was funded by Seaside Therapeutics, the drug's manufacturer, and done jointly by scientists at Rush University Medical Center in Chicago and the MIND Institute at the Universi
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