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A pill that can correct a wide range of faulty genes which cause crippling illnesses should be available within three years, promising a revolution in the treatment of thousands of conditions. The drug, known as PTC124, has already had encouraging results in patients with Duchenne muscular dystrophy and cystic fibrosis. The final phase of clinical trials is to begin this year, and it could be licensed as early as 2009. As well as offering hope of a first effective treatment for two conditions that are at present incurable, the drug has excited scientists because research suggests it should also work...

Close window Published online: 9 November 2006; | doi:10.1038/news061106-16 Drug makes cells ignore mutationExperimental cystic-fibrosis treatment could be used in many diseases.Claire AinsworthA drug that corrects the effects of a genetic mutation has produced encouraging results in tests on patients. The drug, PTC124, is designed to fool a patient's cells into producing a functional protein, even though that protein's gene is mutated. PTC124, made by New-Jersey-based biotech company PTC Therapeutics, is part of a growing field of research targeting therapies at the RNA that ferries information between DNA and proteins. Although results so far are preliminary, they provide proof...