Cystic Fibrosis Therapeutics

摘要: A great deal of excitement and hope has followed the successful trials US Food Drug Administration approval drug ivacaftor (Kalydeco), first therapy available that targets underlying defect causes cystic fibrosis (CF). Although this currently demonstrated a clinical benefit for small minority CF population, developmental pathway established by paves way other transmembrane conductance regulator (CFTR) modulators may many more patients. In addition to investigating CFTR modulators, researchers are actively developing numerous innovative therapies. review, we use catalog treatments under evaluation with support Cystic Fibrosis Foundation, known as Foundation Therapeutics Pipeline, platform discuss variety candidate lung disease promise improve care. Many these approaches target individual components relentless cycle airway obstruction, inflammation, infection characteristic in CF, whereas others aimed directly at gene defect, or resulting dysfunctional protein, instigates cycle. We how new findings from laboratory have informed not only development novel therapeutics, but also rationales their outcomes used measure effects. By reviewing breadth development, well recent progress therapies reflected evolution therapeutics pipeline over past few years, build upon optimism anticipation generated success Kalydeco.