Ataluren Phase 3 Data Encouraging for Cystic Fibrosis

Ataluren, formerly known as PTC124 appears to be a viable treatment for Cystic Fibrosis. This oral compound targets CFTR, the protein product and faulty gene believed to be the cause of Cystic Fibrosis. Ataluren also appears to be particularly effective for patients with Cystic Fibrosis (CF) nonsense mutation. This genetic mutation interrupts the CFTR protein[...]

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