Arbaclofen drug used to treat Fragile X syndrome

September 19, 2012 3:02:54 PM PDT
An experimental drug is offering new hope for treating some behavioral issues associated with Fragile X syndrome as well as autism spectrum disorders.

The medication seems to help balance the brain so patients can overcome social awkwardness such as withdrawal or over excitability.

The drug, called Arbaclofen, is not a cure. But what researchers say it can do it target a genetically induced signaling problem in the brain that makes it hard for these patients to socialize.

It may also solve problems with aggression and sensory overload that can lead to a lot of behavioral issues.

Researchers at Rush University Medical Center have been helping test this compound along with the manufacturer in patients ages 6 to 39 years. They say this is an exciting step because they are seeing improvement.

"Probably not everyone will respond to this medication, but this is a hopeful sign that we are starting to actually develop medications that target the actual brain disorder in autism," said Rush pediatric neurologist Dr. Elizabeth Berry. "And so hopefully we will have a whole list of this in the future and we can treat the brain disorder."

There is more on this preliminary study in the journal Science Translational Medicine.

There are other similar drugs like this in the pipeline. Researchers are hopeful these treatments will be available within the next couple years.


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