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Drug fights cystic fibrosis
Date:2/5/2008

The preclinical and clinical data on PTC124 support our hope that this drug will be an important disease-modifying therapy for cystic fibrosis, said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation in Bethesda, Md. We look forward to the next stage of clinical development to demonstrate the benefits of this promising therapy.

Also in the UAB study, PTC124 was shown to be highly selective for fixing only disease-causing mutations, while it spared normal genes, Bedwell said.

The compound has been granted orphan-drug status by the U.S. Food and Drug Administration for the treatment of Duchenne muscular dystrophy and CF, according to the biopharmaceutical company PTC Therapeutics, Inc. of South Plainfield, N.J. It works in an oral form.


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Contact: Troy Goodman
tdgoodman@uab.edu
205-934-8938
University of Alabama at Birmingham
Source:Eurekalert

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