Addressing Treatment Challenges in Cystic Fibrosis

Program Goals

Pseudomonas aeruginosa

Eradication of Mucoid Pseudomonas Infection

EPIC Clinical Trial*

EPIC Results

Management of Exacerbations

Cystic Fibrosis Guidelines

Cystic Fibrosis Guidelines

Why Don’t Physicians FollowClinical Practice Guidelines?

Personalized Medicine Targeting the CFTR

Strategies for Modifying the Underlying Defect in CF

Strategies for Modifying the Underlying Defect in CF (cont)

Ivacaftor in Patients With CF and the G551D Mutation

F508 (F508del)

Corrector TherapyPhase 2 Results of Lumacaftor + Ivacaftor

Looking to the Future

Will Improving CFTR Function Affect Chronic Pseudomonas

Infection?

Practical Advice to Address the Challenges of Complex Medical

Regimens

Abbreviations

References

References (cont)

References (cont)

References (cont)