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. 2019 Jul 19;365(6450):220-221.
doi: 10.1126/science.aaw0553.

Making precision medicine personal for cystic fibrosis

Candela Manfredi  1 , Janice M Tindall  2 , Jeong S Hong  1 , Eric J Sorscher  3
Affiliations

Making precision medicine personal for cystic fibrosis

Candela Manfredi et al. Science. .
No abstract available

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Figures

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Disease-causing mutation subclasses in cystic fibrosis
There are over 1700 cystic fibrosis (CF) transmembrane conductance regulator (CFTR) mutations, which are classically divided into five categories based on pathogenic mechanism. 39 CFTR mutations are approved by the U.S. Food and Drug Administration for modulator treatment. The proportion of CF patients with at least one mutation in a subclass and the proportion of those patients who can receive modulators is indicated. Figures based on publicly available data (13). * Patients with heterozygous CFTR variants in two classes are counted twice.
See this image and copyright information in PMC

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