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PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirming Activity
Date:6/12/2008

inical benefit of PTC124 in adults and children with nonsense-mutation-mediated CF."

About Cystic Fibrosis

Cystic fibrosis (CF) is among the most common life-threatening genetic disorders worldwide. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and, according to the European Cystic Fibrosis Foundation, it affects a similar number of patients in Europe. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10% of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (http://www.cff.org).

About PTC124

PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce functional protein across a v
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SOURCE PTC Therapeutics, Inc.
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