In July 2012, Pietro Scarso, 3, was diagnosed with Duchenne muscular dystrophy. Most children with this aggressive form of MD are in a wheelchair by age 10 and do not live beyond their teens.
There is no cure for DMD, but Pietro's parents, Dayna and Manni, are determined to extend their son's life.
The Bay Ridge, Brooklyn, family set up pietrosfight.org to raise awareness of DMD and fund research. DMD affects 35 children, mainly boys, per year.
Then they learned about an experimental new drug called Eteplirsen, which is in clinical trials. Manni Scarso said researchers initially were just trying to slow the progression of the disease but after 62 weeks they saw reversal.
The drug is not yet approved by the FDA. The drug maker is scheduled to go before the FDA in mid-February.
The Scarsos are hoping for an accelerated approval so they can start treating Pietro immediately. They hope this drug or any drug will be fast tracked to help save their little boy.
To learn more about DMD or to help Pietro, see: