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New CFTR Modulator treatments

This is an exciting time in the history of Cystic Fibrosis care with development and funding of new treatments. These treatments  tackle the underlying cause of your CF symptoms rather than treating the symptoms themselves.

New CFTR modulator therapies, or precision therapies, which include Kalydeco, Orkambi, Symkevi and Kaftrio all target the effect of your own specific CF mutations on the function of your CFTR protein and help it work more effectively.

Importantly thee treatments only reach approximately 90% of CF cases and are yet not available for some rarer mutations, and will not be tolerated by all. They are also only available currently  in some parts of Europe and the USA which is an inequality for CF friends around the world. That may feel very frustrating. Be reassured that research is ongoing to expand use of CFTR treatments to wider age ranges, different drug combinations and indeed new approaches the bypass the CFTR protein so may allow corrective non mutation specific treatments to be developed – watch this space, or more specifically Sophie’s research page for new trials you may be eligible and be reassured we will be signposting you as things change. The speed of change has been dramatic which can feel a little overwhelming but also exciting and very hopeful for the future.

With new treatments comes uncertainty and new questions. Particularly frequent questions are around ‘can I stop any of my CF treatments ?’. Be reassured that  lots of research is starting in this area so please talk to us before making any changes  and will be listening carefully to results of these trials as we know these questions are very important to you.

Links & Resources


Click here for access to Vertex’s product information for all new CFTR modulator therapies

https://www.cfsource.co.uk/vertex-medicines

 

Click here for access to CF trust page about CFTR modulator treatments and their role in pushing forward access to these treatments

https://www.cysticfibrosis.org.uk/the-work-we-do/campaigning-hard/life-saving-drugs