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Study Shows New Medication Effectively Treats Underlying Cause of Cystic Fibrosis
Date:11/2/2011

SEATTLE, Nov. 2, 2011 /PRNewswire-USNewswire/ -- A new study has confirmed that the drug, ivacaftor (VX-770), significantly improves lung function in some people with cystic fibrosis (CF). The results of the phase III clinical trial study, "A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation," led by Bonnie W. Ramsey, MD of Seattle Children's Research Institute and the University of Washington, were published today in the New England Journal of Medicine.

Ivacaftor, also known as VX-770, was developed by Vertex Pharmaceuticals with financial support from the Cystic Fibrosis Foundation. The oral medicine targets the defective protein produced by the gene mutation called G551D that causes CF.  Researchers found that patients carrying G551D – approximately four percent of all CF patients – who were treated with VX-770 showed a 17 percent relative improvement in lung function that was sustained over the course of 48 weeks.

Additionally, patients with G551D treated with VX-770 showed improvements in other areas critically important to the health of people with CF. Study participants experienced significant reductions in sweat chloride levels indicating an improvement in the body's ability to carry salt in and out of cells – a process which when defective leads to CF. They also experienced decreased respiratory distress symptoms and improved weight gain.  Those who
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SOURCE Seattle Children’s Research Institute
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