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Expert Opin Emerg Drugs. 2007 May;12(2):329-36.

Emerging drug treatments for cystic fibrosis.

Author information

  • The John Hopkins School of Medicine, Department of Pediatrics, Children's Health Building, 200 N. Wolfe St., Baltimore, MD 21287, USA. pzeitlin@jhmi.edu


Although the gene for cystic fibrosis was discovered in 1989, a definitive cure remains elusive for this deadly orphan disease. Advances in nutritional rehabilitation, antibiotics, mucolytics and delivery of care have improved survival to a median age of 37.5 years; however, the psychosocial, personal and financial burdens of this lifelong chronic illness remain considerable. The current portfolio of investigational therapeutics is explored here and placed in a context of therapeutic target and predicted clinical benefit. Partnership between large and small pharma, the Cystic Fibrosis Foundation and academia should be fostered to accelerate therapeutic development.

[PubMed - indexed for MEDLINE]
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