PTC therapeutics awarded 1.6 million from FDA

PTC therapeutics the pharmaceutical maker of Ataluren (PTC124) was recently awarded 1.6 million dollars from the FDA to support ongoing trials of Ataluren (PTC 124) in Duchenne muscular dystrophy.  Ataluren works by targeting a specific type of nonsense mutation or stop codon.  For more on PTC 124 to to research news archives.
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  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

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