LOS ANGELES, May 12 (Xinhua) -- The U.S. Food and Drug Administration (FDA) is convening a meeting of outside experts on Friday to review clinical data on an experimental gene therapy for Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness.
The disease, which almost exclusively affects boys, destroys muscles. Most boys end up in wheelchairs before they become teenagers. Eventually, their hearts and lungs give out. Most people with DMD do not survive beyond their thirties.
The FDA advisers will vote on whether the available data provided by Sarepta Therapeutics Inc. supports accelerated FDA approval for the gene therapy. Then the FDA will decide whether to follow their advice. ■