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Ataluren Phase 2 Data in Nonsense Mutation Cystic Fibrosis Published in the European Respiratory Journal
Date:7/1/2011

forced vital capacity (FVC). At day 84, the aggregate mean change from baseline for all patients was 4.5% and 3.5% for relative percent predicted FEV1 and FVC, respectively. Importantly, combined mean improvements in FEV1 and FVC occurred coincident with the greatest mean improvement in total chloride transport and, with the cessation of ataluren treatment, FEV1 and FVC values reverted towards baseline. The study was not powered to detect statistical significance in these outcome measures and observed changes were not statistically significant.

As an additional secondary endpoint, the study evaluated the frequency of CF-related coughing, which is the most common complaint among patients and often interferes with activities of daily living.(3),(4) During the study, quantitative measurements of CF-related coughing were conducted over a 24-hour period after each clinic visit and results showed that ataluren was associated with an aggregate mean reduction in waking cough frequency of 23% for all patients (p=0.006, paired t-test).

Safety results from the study showed that ataluren was generally well tolerated. Most adverse events were mild or moderate and compliance with study drug was greater than 96% for all patients.

The abstract titled "Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis" is available online at: http://erj.ersjournals.com/content/early/2011/01/13/09031936.00120910.abstract?sid=f34afce6-c9f0-4da2-bcb3-708f931ddda2

ABOUT ATALUREN

An investigational new drug discovered by PTC Therapeutics, ataluren (formerly referred to as PTC124®) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematu
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SOURCE PTC Therapeutics, Inc.
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