, Salem, MA

April 15, 2014

Letter: Drug needs FDA approval

The Salem News

---- — To the editor:

Families of boys with Duchenne muscular dystrophy just filed a petition with more than 100,000 names on the White House website to push approval of the experimental drug Eteplirsen by the Food and Drug Administration.

DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. The disease primarily affects boys. Over time, sometimes as early as the teen years, the damage done by DMD to the heart and lungs can and does become life-threatening.

As a father who watched his son suffer from this hideous disease, I can’t even begin to tell you the pain and suffering this causes to the child, siblings and parents.

Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, who designed the drug trial, said Eteplirsen is a first-of-its-kind therapy that has shown superb results so far and has absolutely no side effects.

The FDA has to use common sense here and forgo any lengthy study, which could take years and cost the lives of thousands who suffer from DMD.

Those affected by DMD need Eteplirsen now, not years from now. Please FDA — do the right thing!

Mark Veilleux