Cystic Fibrosis

Biopharma company presentesresults of trial at cystic fibrosis conference

Published in Gene Therapy Weekly, November 18th, 2004

PTC Therapeutics, Inc., (PTC) announced single-dose phase 1 clinical data for the company's lead candidate, PTC124.

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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Source: Gene Therapy Weekly (2004-11-18)

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