Health

Fragile X Syndrome Experimental Drug To Help Autism Too

Fragile X syndrome or the Martin – Bell syndrome is a genetic condition known as the most common inherited intellectual disability in boys. A new Fragile X syndrome experimental drug has benefits that could help autism patients too.

The Fragile X syndrome is the most common single-gene cause of autism. It is also the most common form of mental retardation passed through inheritance among boys. The syndrome features physical characteristics such as an elongated face or large ears and behavioral characteristics that include social anxiety. A new experimental drug has proved effective in helping Fragile X Syndrome with social withdrawal.

Researchers at the UC Davis MIND Institute and Rush University Medical Center in Chicago found most likely the first Fragile X syndrome experimental drug to be effective in patients’ need for social withdrawal. The new drug, called Arbaclofen or compound STX 209, makes people with Fragile X syndrome or autism to be less likely in avoiding social interactions.

“This study shows that STX 209 could become an important part of the treatment for fragile X syndrome, because it appeared to improve symptoms in those with significant social deficits or autism” explains Randi Hagerman, director at MIND Institute. Since until now there wasn’t any targeted treatment for autism, Arbaclofen or compound STX 209 could become the first drug to do just that, adds Hagerman.

“This study nails a core feature in autism. We think is a great drug” Randi Hagerman tells Los Angeles. This means that at least the 2 percent autism cases caused by Fragile X syndrome could really benefit from this new experimental drug, let alone the actual patients with Fragile X.

“It would be, I think, unrealistic to expect that this drug would be uniformly beneficial to all people that have an autism diagnosis” Mark Bear, cofounder of Seaside Therapeutics, the maker of Arbaclofen, said. “But I think we can still be quite optimistic that it can be beneficial to a subset of those patients” he added.

In a nutshell, the new Fragile X syndrome experimental drug looks like the milestone in the treatment research for autism too. Arbaclofen offers hope not only to Fragile X syndrome patients, but to many of the children or adults with autism and their caregivers.

“Andy is now 23 years old and we are so eager to get him started in one of these trials” says Katie Clapp whose son was born with the Fragile X syndrome.

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