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Vertex wins broader use of Kalydeco in CF

This article was originally published in Scrip

Executive Summary

Vertex Pharmaceuticals on 21 February won approval from the FDA to expand the use of Kalydeco (ivacaftor) for use in eight additional mutations that cause cystic fibrosis, a rare, life-threatening genetic disease caused by a defective or missing CFTR protein, resulting from mutations in the CFTR gene, which causes poor flow of salt and water into and out of the cell in a number of organs, including the lungs.

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