Future Treatments, in Cystic Fibrosis (Oxford Respiratory Medicine Library) (2010).

Griesenbach U, Davies JC, Alton EW

Oxford University Press

• Increased understanding of CF pathophysiology is leading to new therapies
• Several gene therapy trials have established proof-of-principle for gene transfer in the airways, but a gene therapy-based treatment has not yet been developed
• Small molecule drugs directed at class-specific CFTR mutations are showing promise in early phase trials
• Drugs aimed at restoring hydration of the airway surface have been shown to improve mucociliary clearance
• Increasing numbers of antibiotics are being formulated for both nebulization and inhalation.