PORTLAND, Maine - For the first time, the federal Food and Drug Administration has approved a drug to treat Duchenne muscular dystrophy.
Brian Denger, of Biddeford, says he's ecstatic that the drug eteplirsen might soon be available for his 22-year-old son, Patrick, who has the rare genetic disorder that causes progressive muscle weakness.
"It's not a cure. It won't reverse the course of the disease," Denger says. "But it's possible that it could slow the progression, so that he could retain the things he still continues to do."
Denger traveled to Maryland last spring to lobby for the approval of eteplirsen. The FDA's approval goes against the recommendation of an advisory committee that said there was insufficient evidence of the drug's effectiveness. The FDA is requiring further clinical trials to confirm clinical benefit.
