Ryleigh Messmer is a 15-year-old from Colorado Springs who has cystic fibrosis (CF), a rare, life-shortening genetic disease that affects the lungs, pancreas, and other organs. When she was born, the only treatments to maintain lung health for people with CF were mucus clearance treatments to try to alleviate symptoms such as the buildup of thick, sticky mucus that would eventually damage the lungs.
However, just two years later everything changed when the FDA approved ivacaftor, the first drug designed to target the defective protein that causes CF. This protein is called the cystic fibrosis transmembrane conductance regulator or CFTR. Much of the clinical research that ultimately led to the approval of ivacaftor, the first CFTR modulator, in 2012 took place at Children’s Hospital Colorado in collaboration with the Colorado Clinical and Translational Sciences Institute (CCTSI), which provides specialized research nursing, expert laboratory services and clinical space for the hundreds of children who participated in the research studies over the years…
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