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Ataluren Phase 3 trial results in nonsense mutation cystic fibrosis
Date:5/16/2014

s study were CF pulmonary exacerbations and were considered unrelated to ataluren treatment. Eight patients in the ataluren arm and three patients in the placebo arm discontinued treatment due to an adverse event.

"We are very encouraged by the data from this trial. Given spirometry and pulmonary exacerbation results in the subgroup of patients not receiving chronic inhaled tobramycin, and a favorable safety profile, this study supports further clinical testing of ataluren as a potential first-in-class treatment for nmCF patients not receiving chronic inhaled tobramycin," stated Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, Inc. "We look forward to initiating a confirmatory ataluren trial in nmCF patients later this year."


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Contact: Katelyn McCarthy
katelyn.mccarthy@uhhospitals.org
216-767-8582
University Hospitals Case Medical Center
Source:Eurekalert

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