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A new drug may help certain patients with cystic fibrosis



Scientific research continues to develop novel treatments for patients who have chronic diseases, and these medications may eventually become available at a Canadian internet pharmacy.

Scientific research continues to develop novel treatments for patients who have chronic diseases, and these medications may eventually become available at a Canadian internet pharmacy. One such illness is cystic fibrosis (CF), a genetic disorder in which the body produces abnormally thick mucus that can lead to infection and damage to the lungs and pancreas. Researchers recently completed a phase III clinical trial on a new drug that, if approved, may help certain CF patients who carry a specific mutation.

A team of scientists from the Seattle Children's Research Institute and the University of Washington studied the drug ivacaftor (VX-770), in CF patients whose disease is attributed to a mutation known as G551D. This variation accounts for 4 percent of all CF cases.

The phase III trial included 161 patients, aged 12 years or older, who carried at least one defective G551D copy and took at least one dose of ivacaftor or placebo. Results showed that subjects in the treatment group experienced a 17 percent improvement in their lung function over 48 weeks, as published in the New England Journal of Medicine. These patients also experienced other improvements in their condition, including better weight gain, less respiratory stress and a lower salt content in their sweat.

"Our study shows that we are now able to improve the quality of life for cystic fibrosis patients with the G551D mutation with the administration of VX-770," said researcher Bonnie Ramsey, M.D.

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