1. Advances in Cystic Fibrosis 2017

2. This program will include a discussion of investigational agents not approved by the FDA for use in the US, and data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

3. Cystic Fibrosis

4. Treatment Landscape of Cystic Fibrosis

5. Multidisciplinary Team Approach

6. Preview of What's to Come

7. Advances in Modulator Therapies Targeting Common and Uncommon CFTR Mutations

8. Previous CFTR Modulators Potentiators

9. Previous CFTR Modulators Correctors

10. TEZ/IVA for F508del Homozygous Patients

11. TEZ/IVA for F508del Heterozygous and Residual Function Mutation

12. TEZ/IVA Efficacious and Well Tolerated in F508del Heterozygous Population

13. Closing Comments

14. Novel Approaches Targeting CFTR Mutations

15. CFTR Correctors

16. GLPG2222 Well Tolerated

17. The Potentiator GLPG2451 With and Without the CFTR Corrector GLP2222 in Healthy Volunteers

18. A Novel Type of Modulator Called an Amplifier

19. The Novel CFTR Amplifier PTI-428 Shows Promise

20. Nebulized QR-010: A Different Approach to Restore Mutant CFTR Function Using Oligonucleotides

21. Summary

22. Nursing Perspective on Quality of Life and Disease Burden

23. QoL After 3 Months of Ivacaftor -- Children's Hospital, Philadelphia

24. Results

25. Takeaways

26. Caregiver Burden Due to Pulmonary Exacerbations in CF

27. Findings

28. Concluding Remarks

29. Longer Term and Real-Life Outcome Data

30. We Know the Effects of FEV1 -- but What Is the Mechanism?

31. Effect of LUM/IVA on Total Bronchiectasis and Air Trapping CT Scores in Children: F508del-CFTR Mutation

32. Safety and Efficacy of LUM/IVA in Patients With CF Homozygous for F508del-CFTR

33. Effectiveness of LUM/IVA in Patients With F508del Homozygous CF Following FDA Approval

34. Modeling Long-Term Health Outcomes in Patients With CF Homozygous F508del Treated With LUM/IVA

35. Concluding Remarks

36. Abbreviations

37. Abbreviations (cont)