Groundbreaking treatment for patients with cystic fibrosis

Treatment with two medications that target the most common genetic cause of cystic fibrosis improves lung function and lowers the rate of pulmonary exacerbations, according to the results from a Phase III international clinical trial published online in the New England Journal of Medicine on May 17, 2015. Combined lumacaftor-ivacaftor therapy was shown to be safe and effective for cystic fibrosis patients with two copies of the cystic fibrosis gene mutation (F508del) found in nearly half of the ...