Muscular dystrophy is a group of genetic disorders that cause the muscles in the body to steadily weaken over time and mobility to be lost. It reduces life span and there is currently no cure. DMD is the commonest and most severe form. There are treatments that can help lessen symptoms, such as muscle spasm, and enable people with MD to lead a good quality of life. Scientist hope that the PTC124 was able to restore the muscle function normally lost in Duchenne Muscular Dystrophy disease. The drug works by allowing cells to read through certain mistakes in the genetic code for a protein - dystrophin - missing in 15% of patients with DMD.

Scientist feel that this drug appears to have the potential to become a treatment in the longer term for some people with DMD.
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