Ivacaftor treatment of cystic fibrosis patients with the G551D mutation: a review of the evidence.

@article{Kotha2013IvacaftorTO,
  title={Ivacaftor treatment of cystic fibrosis patients with the G551D mutation: a review of the evidence.},
  author={Kavitha Kotha and John P Clancy},
  journal={Therapeutic advances in respiratory disease},
  year={2013},
  volume={7 5},
  pages={288-96}
}
Cystic fibrosis (CF) is a recessive disorder caused by mutations in the gene that encodes the CF transmembrane conductance regulator (CFTR) protein. CFTR protein is a chloride and bicarbonate channel that is critical for normal epithelial ion transport and hydration of epithelial surfaces. Current CF care is supportive, but recent breakthroughs have occurred with the advent of novel therapeutic strategies that assist the function of mutant CFTR proteins. The development and key clinical trial… CONTINUE READING

From This Paper

Figures, tables, and topics from this paper.
8 Citations
54 References
Similar Papers

Citations

Publications citing this paper.
Showing 1-8 of 8 extracted citations

References

Publications referenced by this paper.
Showing 1-10 of 54 references

Similar Papers

Loading similar papers…