Children’s Hospital of Pittsburgh of UPMC Only Center in Pennsylvania Participating in Research Study of New Cystic Fibrosis Treatment
PITTSBURGH, July 12 – Children’s Hospital of Pittsburgh of UPMC is the only site in Pennsylvania taking part in a Phase III clinical trial of an investigational drug, Ataluren, to treat nonsense mutation cystic fibrosis (nmCF). Cystic Fibrosis (CF) is a life-threatening genetic disease that causes serious lung infections and digestive complications. Patients with CF lack adequate levels of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is necessary for normal function of the lung, pancreas, liver and other organs. In nmCF, an