Emerging drug treatments for cystic fibrosis

Pamela L. Zeitlin

Research output: Contribution to journalReview article

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.

Original languageEnglish (US)
Pages (from-to)329-336
Number of pages8
JournalExpert Opinion on Emerging Drugs
Volume12
Issue number2
DOIs
StatePublished - May 1 2007

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

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

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