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Another science post too important not to be in the open forum.
The FDA has granted fast track and orphan drug designation to PTC124. It enters the next stage of human trials in Q4 2007/Q1 2008.
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PTC site link....
The FDA has granted fast track and orphan drug designation to PTC124. It enters the next stage of human trials in Q4 2007/Q1 2008.
Story Link....
PTC site link....
Superpill PTC124 Could Potentially Cure 2,000 Diseases
Researchers in the United States have developed a mind-blowing new pill, known as PTC124, that could one day treat nearly 2,000 inherited diseases, including muscular dystrophy and cystic fibrosis. Even better yet, scientists believe that this new "superpill" could be available within just three years.
The way that the drug works is by forcing the body to ignore genetic mutations and produce normal proteins, rather than the mutated versions that lead to disease. In order for it to be effective, patients would need to take it for the entire course of their lives.
Following the promising results revealed in mice, PTC124 is already in the early stages of human trials. And if the trials go well, scientists say that it could be licensed as early as 2009.
PTC124 is unique in that it can correct mutations in almost 2000 diseases and illnesses. It doesn't just target one mutation, but rather, a whole class of them.
The pill is produced by PTC Therapeutics, and in a study published today in Nature Magazine, it shows that in mice with a mutation that causes Duchenne muscular dystrophy the drug starts dystrophin production and restores muscles back to full health.
Researchers in the United States have developed a mind-blowing new pill, known as PTC124, that could one day treat nearly 2,000 inherited diseases, including muscular dystrophy and cystic fibrosis. Even better yet, scientists believe that this new "superpill" could be available within just three years.
The way that the drug works is by forcing the body to ignore genetic mutations and produce normal proteins, rather than the mutated versions that lead to disease. In order for it to be effective, patients would need to take it for the entire course of their lives.
Following the promising results revealed in mice, PTC124 is already in the early stages of human trials. And if the trials go well, scientists say that it could be licensed as early as 2009.
PTC124 is unique in that it can correct mutations in almost 2000 diseases and illnesses. It doesn't just target one mutation, but rather, a whole class of them.
The pill is produced by PTC Therapeutics, and in a study published today in Nature Magazine, it shows that in mice with a mutation that causes Duchenne muscular dystrophy the drug starts dystrophin production and restores muscles back to full health.
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