Article
Entering the era of highly effective modulator therapies.
Pediatric pulmonology - 1 Feb 2021
Dave Kavita, Dobra Rebecca, Scott Sandra, Saunders Clare, Matthews Jess, Simmonds Nicholas J, Davies Jane C
Abstract excerpt
Since the discovery of the gene responsible for cystic fibrosis (CF) in 1989, hopes have been pinned on a future with novel therapies tackling the basis of the disease rather than its symptoms. These have become a reality over the last decade with the development through to the clinic of CF transmembrane conductance regulator (CFTR) modulators. These are oral drugs which improve CFTR protein function through...
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