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Cystic Fibrosis Ireland

Research e-Zine May 2018

Thank you for signing up to receive a copy of the Cystic Fibrosis Ireland Research e-Zine.

We hope you will agree that it’s an extremely exciting time for cystic fibrosis (CF) research with many new and innovative CF treatments being developed or approved.

This e-Zine, similar to the eCystic Fibrosis Review, published by Johns Hopkins University, operates like a “journal club”, providing a concise review of the relevant journal literature on specific topics within CF research.

This issue is split into three sections:

1: Glossary & Brief Overview of the Genetics of CF

2: Safety, Efficacy and Real World Data on Kalydeco and Orkambi

3: Future CFTR Modulator Therapies and Personalised and Predictive Diagnostics
1: Glossary & Brief Overview of the Genetics of CF
In the first section, we provide you with an overview of some of the key definitions and statistical concepts which you might find useful in understanding CF Clinical research. We also provide you with a brief overview of the genetics of CF and the six broad classes of CF causing mutation.
2: Safety, Efficacy and Real World Data on Kalydeco and Orkambi
In the second section, we discuss the safety and efficacy of existing CFTR modulator drugs Kalydeco and Orkambi. These groundbreaking drugs have shown sustained improvements for CF patients not only in clinical trials but through “real-world” settings.
3: Future CFTR Modulator Therapies and Personalised and Predictive Diagnostics
In the third section, we cover new CFTR modulator drug therapies. This includes promising initial results of Tezacaftor/Ivacaftor formulations and triple combination therapies which offer hope to CF patients with minimal function mutations (where little of no CFTR protein is produced) who currently have no pre-existing drug therapies available to them.

Finally, we look at the potential for predictive diagnostics to be used to extend the use of existing CFTR modulator drug therapies, such as Kalydeco, to those with rare mutations.
Copyright © 2018 Cystic Fibrosis Ireland, All rights reserved.


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