A magic bullet that could treat cystic fibrosis, muscular dystrophy, hemophilia and more than 1,800 other genetic disorders could be available by 2009. Lee Sweeney of UPenn, leader of the team developing the drug, tells the Times of London: “It doesn’t just target one mutation that causes disease, but a whole class of mutations.”
The breakthrough drug, known as PTC124, works by helping genes to ignore disruptions to genetic code caused by "nonsense mutations," allowing cells to develop normally. It can be used against a wide range of disorders caused by the same kind of mutation. The daily pill enters the last phase of trials on humans this year.