Times article on Fragile X clinical trial

There’s a very nice article in the New York Times today [Promise Seen in Drug for Retardation Syndrome] about a human clinical trial testing a drug to improve behavior in those with Fragile X syndrome (FXS), a genetic disorder associated with intellectual disability and behavior problems, among other issues.  The article traces the long path that led to the development of drugs for FXS.   The results of the clinical trial are not yet published and larger studies will surely be needed, but there is new hope that pharmaceutical interventions may be able to correct some of the alterations that occur in the brain of those with FRAX.  The article also mentions the FRAXA Research Foundation, which has been key in advancing research for Fragile X.  Founded in the ‘90s by three very motivated parents who were frustrated with how little research was being done to find new therapies for their children’s disorder, FRAXA has been at the forefront of engaging the research community and supporting promising research on this disorder.  Kudos to them for providing a wonderful example of how a small group of motivated individuals can make a huge impact.