News Headlines Article

A Family’s Long Search For Fragile X Drug Finds Frustration, Hope
National Public Radio

For a few weeks last year, Michael Tranfaglia and Katie Clapp saw a remarkable change in their son, Andy, who’d been left autistic and intellectually disabled by fragile X syndrome. Andy, who is 25, became more social, more talkative and happier. “He was just doing incredibly well,” his father says.

The improvements came while Andy was taking an experimental drug — a drug made possible by the efforts of his parents. And at the time, it appeared this drug might become the first effective treatment for fragile X, a rare genetic syndrome that affects brain development and can lead to intellectual and social disabilities. The reality would turn out to be more complicated.