Advances in Cystic Fibrosis 2017

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.

Cystic Fibrosis

Treatment Landscape of Cystic Fibrosis

Multidisciplinary Team Approach

Preview of What's to Come

Advances in Modulator Therapies Targeting Common and Uncommon CFTR Mutations

Previous CFTR Modulators Potentiators

Previous CFTR Modulators Correctors

TEZ/IVA for F508del Homozygous Patients

TEZ/IVA for F508del Heterozygous and Residual Function Mutation

TEZ/IVA Efficacious and Well Tolerated in F508del Heterozygous Population

Closing Comments

Novel Approaches Targeting CFTR Mutations

CFTR Correctors

GLPG2222 Well Tolerated

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

A Novel Type of Modulator Called an Amplifier

The Novel CFTR Amplifier PTI-428 Shows Promise

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

Summary

Nursing Perspective on Quality of Life and Disease Burden

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

Results

Takeaways

Caregiver Burden Due to Pulmonary Exacerbations in CF

Findings

Concluding Remarks

Longer Term and Real-Life Outcome Data

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

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

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

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

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

Concluding Remarks

Abbreviations

Abbreviations (cont)