Medical scientists at PTC Therapeutics (S. Plainfield, NJ) have entered into trials an oral drug therapy aimed at treating Duchenne muscular dystrophy and other genetic conditions caused by “nonsense mutations.” Nonsense mutations inactivate gene function and are known to cause anywhere from five to 70% of the individual cases of most inherited diseases, such as cystic fibrosis (10%) and Hurler’s syndrome (70%). The drug, PTC124, is designed to bypass the mutations.
“As these preclinical data demonstrate, the broad potential of PTC124 lies in its specificity and unique mechanism of action, which has the potential to address the underlying cause of a broad range of genetic disorders due to nonsense mutations,” said Stuart W. Peltz, PhD, president and CEO of PTC Therapeutics.
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