Abstract
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.
MeSH terms
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Animals
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Chloride Channel Agonists
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Chloride Channels / genetics
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Chloride Channels / metabolism
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Cystic Fibrosis / drug therapy*
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Cystic Fibrosis / genetics
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Cystic Fibrosis / metabolism
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Cystic Fibrosis Transmembrane Conductance Regulator / agonists
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Cystic Fibrosis Transmembrane Conductance Regulator / genetics
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Cystic Fibrosis Transmembrane Conductance Regulator / metabolism
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Drugs, Investigational / pharmacology
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Drugs, Investigational / therapeutic use*
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Humans
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Oxadiazoles / pharmacology
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Oxadiazoles / therapeutic use
Substances
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Chloride Channel Agonists
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Chloride Channels
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Drugs, Investigational
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Oxadiazoles
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Cystic Fibrosis Transmembrane Conductance Regulator
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ataluren