Fox 5 News Leaks SRPT Date with FDA For Early Approval. LINKED.
osted: Jan 31, 2013 5:22 PM EST Updated: Feb 01, 2013 9:36 AM EST
By ADRIENNE SUPINO, Fox 5 News Reporter
NEW YORK (MYFOXNY) -
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.
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.