Current and Emerging Therapies for the Treatment of Cystic Fibrosis or Mitigation of Its Symptoms
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1.6 BIOLOGICAL SCIENCES, 3. MEDICAL AND HEALTH SCIENCES
Clinical presentation of the chronic, heritable condition cystic fibrosis (CF) is complex, with a diverse range of symptoms often affecting multiple organs with varying severity. The primary source of morbidity and mortality is due to progressive destruction of the airways attributable to chronic inflammation arising from microbial colonisation. Antimicrobial therapy combined with practises to remove obstructive mucopurulent deposits form the cornerstone of current therapy. However, new treatment options are emerging which offer, for the first time, the opportunity to effect remission from the underlying cause of CF. Here, we discuss these therapies, their mechanisms of action, and their successes and failures in order to illustrate the shift in the nature of how CF will likely be managed into the future.
Murphy, M.P. & Caraher, E. (2016). Current and Emerging Therapies for the Treatment of Cystic Fibrosis or Mitigation of Its Symptoms. Drugs in R & D, vol. 16, no. 1, pg. 1–17. doi: 10.1007/s40268-015-0121-9