Published in Gene Therapy Weekly, November 18th, 2004
PTC124 is a small-molecule drug being investigated initially as a treatment for a subset of patients with cystic fibrosis and Duchenne muscular dystrophy due to nonsense mutations, with the potential to treat a number of other genetic disorders. The results were presented at the 18th Annual North American Cystic Fibrosis Conference in St. Louis, Missouri.
The presentation included results from a phase 1 single-dose study that was designed to evaluate the drug's safety, palatability, and pharmacokinetic profile across escalating doses in...
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