Ivacaftor: a novel mutation modulating drug.

  title={Ivacaftor: a novel mutation modulating drug.},
  author={Harit Kapoor and Astha Koolwal and Ankur Singh},
  journal={Journal of clinical and diagnostic research : JCDR},
  volume={8 11},
Cystic fibrosis (CF) is multisystemic disorder presenting in newborn period to adulthood, predominantly affecting respiratory system. It is caused by mutation in CF transmembrane conductance regulator gene. ΔF508 is the most common mutation seen worldwide. Supportive management with bronchodilators, anti-inflammatory, mucolytics, antibiotics are the corner stone of therapy. Mutation specific drug, Ivacaftor, was recently approved USFDA in January 2012 for patients carrying G551D mutation. It is… CONTINUE READING