@article{5c85d7a03950487fab3963bc54c7198a,
title = "Emerging drug treatments for cystic fibrosis",
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.",
keywords = "Absorption, Calcium-activated chloride channel, Chloride, ClC-2, Cystic fibrosis transmembrane conductance regulator, Endoplasmic reticulum, Epithelia, Gene therapy, Inositol polyphosphate, Ion channel, Mannitol, Mucolytic, Plasma membrane, cAMP",
author = "Zeitlin, {Pamela L.}",
note = "Funding Information: Current estimates project that it requires US$800 million to take an investigational agent from concept to market. Given the declining availability of funding available from the US National Institutes of Health to support basic and clinical research, the limited resources of small pharma and biotech startups, and the small market presented by an orphan disease, special incentives are needed to fuel and maintain drug development for CF. Cystic Fibrosis Foundation Therapeutics (CFFT) was founded in 2000 as a non-profit, drug discovery and development affiliate of the Cystic Fibrosis Foundation. CFFT provides matching research awards to scientists in academia and the pharmaceutical industry who then gain access to a specialized network of CF clinical research centers. These competitive grants support the drug discovery phase through several stages of evaluation to complete the full length of the drug development pipeline. A specialized network of CF clinical research centers with strong basic laboratory research components was founded in 1998 (Cystic Fibrosis Therapeutics Development Network). This network specializes in technically complex surrogate outcome measures for Phase I and II trials and has facilitated the progress of a variety of therapeutics along the drug development pipeline [60].",
year = "2007",
month = may,
doi = "10.1517/14728214.12.2.329",
language = "English (US)",
volume = "12",
pages = "329--336",
journal = "Expert Opinion on Emerging Drugs",
issn = "1472-8214",
publisher = "Informa Healthcare",
number = "2",
}