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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.