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: