Nationwide clinical drug trials give hope for patients with autism and fragile X syndrome

As scientists uncover the precise links between autism and the fragile x gene, potential treatments to reverse many of the core symptoms of both autism and fragile X syndrome (FXS) are moving closer to reality.  Nationwide clinical drug trials are already underway and are showing significant success in reversing symptoms of severe social impairment and behavioral issues in participants with autism and FXS, giving hope to families and individuals with these conditions that additional treatments may be on the horizon.  

"For the first time, we have hope that medicines are within sight that will allow many individuals with fragile X syndrome and autism, who have severe social impairments, to be able to function and interact with others in society," said Robert Miller , Executive Director of the National Fragile X Foundation. 

While autism is the more commonly known of the two conditions, fragile X syndrome is the most common known genetic cause of autism and autism spectrum disorders.  It is also the most common cause of inherited intellectual disability and can cause learning and behavioral disorders that can range from mild to severe.  New scientific research has further established the interconnectivity between these two conditions.

The enthusiasm for these new discoveries into the genetic causes of autism is tempered by the reality that federal budget constraints may jeopardize the significant scientific gains made.  Hundreds of advocates, including researchers and families of individuals with FXS and autism, will be in Washington on March 5 and 6 to help raise public and Congressional awareness of the break-through research's potential for treatment. 

"With actual treatments for key symptoms of autism and fragile X syndrome in the testing phase, the impact of recent scientific discoveries cannot be underestimated—and should not be starved of support just as they are coming to reality," Miller said.