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Cystic Fibrosis Foundation Issues Statement on The New England Journal of Medicine Study Showing Evidence of Success in Treating Underlying Cause of Cystic Fibrosis

November 2, 2011

(Bethesda, Md.) – The Cystic Fibrosis Foundation issued the following statement in response to study results published today by The New England Journal of Medicine offering first evidence that the underlying cause of cystic fibrosis in select patient groups can be treated through a new drug, Kalydeco™ (VX-770).

The New England Journal of Medicine study marks an important and exciting milestone for the cystic fibrosis community, and it confirms that we are one step closer to finding a cure for the disease,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Nearly every CF drug available today was made possible with Foundation support, and Vertex Pharmaceuticals’ Kalydeco is the latest example of our venture philanthropy model at work. We are excited about the opportunity this treatment presents if approved by the FDA for those CF patients who can benefit from this new drug.”

Kalydeco, developed by Vertex Pharmaceuticals with CF Foundation support and research input, is the first CF drug to correct a specific genetic defect — mutation G551D — in select CF patient groups. The drug is currently under review for approval by the U.S. Food and Drug Administration. For more information, click here. To view The New England Journal of Medicine study and editorial, click here.

About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, go to www.cff.org.

The Cystic Fibrosis Foundation is an accredited charity of the Better Business Bureau.