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PTC Therapeutics Announces $25 Million Award From Cystic Fibrosis Foundation Therapeutics for Development of PTC124
Date:7/16/2008

data from recently completed Phase 2a studies in adult and pediatric CF patients at the European Cystic Fibrosis Conference. Results from these studies demonstrated statistically significant improvements in the primary outcome measure-nasal potential difference (NPD)-across a variety of age ranges, geographies, and nonsense mutation types. In the three-month, Phase 2a extension study of adult patients, results demonstrated a statistically significant mean decrease [28 percent] in the frequency of cough, one of the most prominent and burdensome CF-related symptoms.

About Cystic Fibrosis

Cystic fibrosis (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. CF occurs in approximately one of every 3,500 live births, with approximately 1,000 new cases diagnosed each year in the United States. 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 percent 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 CF patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments for the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation ( '/>"/>

SOURCE PTC Therapeutics, Inc.
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