Newer therapies for cystic fibrosis.
Cystic fibrosis (CF) is a chronic, progressive, genetic disease caused by flawed ion transport across epithelial membranes due to a genetic mutation. Most therapeutic efforts are centred on the main clinical manifestations of the disease: progressive destructive airway disease and pancreatic insufficiency. Most individuals with CF succumb to lung disease. The present-day therapeutic armamentarium includes agents that have been used for many decades, some of which have experienced transformations in their formulation or mode of administration thanks to the introduction of new manufacturing technologies. The development of new therapies involves new conceptual approaches, based on recent understanding of the disease. These therapies await proof of concept or clinical experimentation before being accepted as useful means to arrest the progression of the disease. In this article we will review therapeutic agents introduced into the clinical arsenal in the last 20 years, as well as experimental therapies under active investigation.
Schidlow
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Department of Pediatrics, MCP Hahnemann University School of Medicine, St. Christopher's Hospital for Children, Erie Avenue at Front Street, Philadelphia, PA 19134-1095, USA. daniel.schidlow@drexel.edu
D000900:Anti-Bacterial Agents / Q000627:therapeutic use*; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D001424:Bacterial Infections / Q000188:drug therapy*; D003550:Cystic Fibrosis / Q000188:drug therapy / Q000601:surgery / Q000628:therapy*; D019587:Dietary Supplements; D015316:Genetic Therapy; D006801:Humans; D016040:Lung Transplantation
D000900:Anti-Bacterial Agents; D000893:Anti-Inflammatory Agents