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PTC Therapeutics Initiates Registration-Directed Phase 3 Trial of Ataluren in Cystic Fibrosis
Date:9/10/2009

rders due to a nonsense mutation. Restoring functional CFTR protein may enable ataluren to address the underlying cause of nmCF and change the course of the disease. Currently available therapies for CF focus only on the alleviation of symptoms.

"We are excited to expand the ataluren registration program to include nmCF, an area of significant unmet medical need," said Langdon Miller, M.D., Chief Medical Officer of PTC Therapeutics. "We are hopeful that this Phase 3 trial together with our ongoing studies in nmDMD/BMD, will add to a growing body of data demonstrating ataluren's potential for treatment of patients with nonsense mutation genetic disorders."

About Cystic Fibrosis (CF)

CF is a life-threatening genetic disease that causes serious lung infections and digestive complications. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and nearly 70,000 people worldwide. Genetic testing is required to confirm a complete diagnosis and to determine if a patient's disease is caused by a nonsense mutation. It is estimated that nonsense mutations are the cause of CF in 10 percent of patients in the United States and Europe and over 50 percent of patients in Israel. Available treatments for CF are designed to alleviate symptoms rather than correct the underlying cause of the disease. These treatments include chest physical therapy to clear 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. More information regarding CF is available through the Cystic Fibrosis Foundation (www.cff.org).

About Atal
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SOURCE PTC Therapeutics, Inc.
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