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Anaheim to Host World’s Largest Gathering on Cystic Fibrosis Research and Care

First Potential Drug to Treat Underlying Cause of Fatal Disease under Review by FDA

November 1, 2011

(Bethesda, Md.) –  For the first time, a drug that’s designed to treat the underlying cause of cystic fibrosis, Kalydeco™ (ivacaftor), is under review by the U.S. Food and Drug Administration for possible approval in 2012. This milestone comes as the largest international gathering of cystic fibrosis experts kicks off in Anaheim, Calif., Nov. 3 – 5, 2011.

Kalydeco (kuh-LYE-deh-koh) is designed to address the genetic defect in cystic fibrosis in select patient groups, unlike all CF therapies now on the market, which only treat symptoms of the disease. It was formerly known as VX-770.

At the 25th annual North American Cystic Fibrosis Conference (NACFC), physicians, scientists and clinicians will present data and workshops on the latest advancements in CF related to research, care and drug development, including new clinical data on Kalydeco.

Key conference topics include:

  • Emerging therapies that target the core defect in CF.
  • Partnering with patients and families to improve care.
  • Best practices and mentoring for adult CF care providers.
  • Effective treatment and management of lung infections.

Cystic fibrosis is a genetic disease that causes life-threatening lung infections and premature death. An estimated 30,000 people in the United States, and about 70,000 people worldwide, have CF. Ten million Americans are symptomless carriers of a defective CF gene. Fifty years ago, most children with CF died before reaching kindergarten. Today, because of Cystic Fibrosis Foundation-supported drug research and care, many people with CF are living into their 30s, 40s and beyond.

WHAT:

The 25th Annual North American Cystic Fibrosis Conference, the largest international gathering of cystic fibrosis research scientists and clinicians.

WHEN & WHERE: 

Thursday, Nov. 3 – Saturday, Nov. 5, 2011, at the Anaheim Convention Center, Anaheim, California.

KEYNOTE SPEAKERS:

Thursday, Nov. 3, 4:20 p.m. – Marcus A. Mall, M.D., University of Heidelberg, Heidelberg, Germany: “CFTR Modulation – 25 Years of NACFC Progress.”

Friday, Nov. 4, 9:00 a.m. – Garry R. Cutting, M.D., and Patrick R. Sosnay, M.D., Johns Hopkins University, Baltimore, Maryland: “CFTR2 – A Research and Clinical Practice Tool.”

Saturday, Nov. 5, 9:00 a.m. – Patrick A. Flume, M.D., Medical University of South Carolina, Charleston, South Carolina: “Pulmonary Exacerbations.”

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.

Media Contact

  • Laurie Fink, national director of media relations, Cystic Fibrosis Foundation: (301) 841-2602; lfink@cff.org.
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The Cystic Fibrosis Foundation is an accredited charity of the Better Business Bureau.