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Normal cells assemble amino acids into protein molecules essential for good health according to a "recipe" encoded into the DNA. In about 15 per cent of patients with DMD, a DNA "nonsense mutation" causes the cells to stop making the protein chains prematurely, rendering the proteins useless.
PTC124 fixes this by prompting the cells to ignore the stop mutations that tell them to end the protein chain, allowing themto complete the correct sequence.
Associate Professor Kornberg said caution was still required because treatments that worked in mice sometimes turned out not to work in humans. "Having said that, the theory behind PTC124 is better than we have ever got to before," he said.
New drug may cure genetic diseases 
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