Cystic Fibrosis: New Insights 25 Years After Gene Discovery
Cystic Fibrosis Foundation:
CF is a life-threatening genetic disease that primarily affects the lungs and digestive system. An estimated 30,000 children and adults in the United States (70,000 worldwide) have CF
CFTR Potentiators: Not an Open and Shut Case.
John P. Clancy, Science Translational Medicine, July 23, 2014
John P. Clancy, Science Translational Medicine, July 23, 2014
The $300,000 Drug. "An excellent example of the promise of personalized medicine", but at what cost?
Joe Nocera, New York Times, July 20, 2014
Identification of the cystic fibrosis gene: Chromosome walking and jumping.
Rommens JM et al. Science (1989)
Rommens JM et al. Science (1989)
The cystic fibrosis gene: A molecular genetic perspective.
Lap-Chee Tsui et al. Cold Spring Harbor, Perspectives in Medicine (2013)
Lap-Chee Tsui et al. Cold Spring Harbor, Perspectives in Medicine (2013)
Neonatal cystic fibrosis screening,
Medline Plus, May 2014
Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis
Cholon D, et al. Sci Transl Med 23 July 2014: Vol. 6, Issue 246, p. 246ra96
Cholon D, et al. Sci Transl Med 23 July 2014: Vol. 6, Issue 246, p. 246ra96
Cystic fibrosis drug combo may be less effective than hoped,
Medline Plus, Jul 23
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