In this session, Professor Jane Davies, will describe the progress and the promise of highly effective CFTR modulator therapies for people with CF. She will highlight the long-term benefits such therapy has had on the relatively small proportion of the CF community for whom a highly effective modulator has been available for several years -- those with the gating mutation G551D who are on ivacaftor -- and use this evidence to describe the impact we might expect from a triple-combination drug in a larger population. The additional opportunities posed by introducing CFTR functional restoration in early life will be illustrated with pre-school and infant data. Whilst celebrating the success of highly effective modulator therapy, she will also discuss some of the challenges that remain, including developing treatments for people with rarer mutations and accessing CFTR modulators in certain regions of the world.
Summarize the progress to date in CFTR modulator development, including the recent successful Phase 3 trials of triple-combination therapies for people with either a single copy of the F508del mutation (heterozygous) or two copies of F508del (homozygous).
Illustrate the long-term benefits of highly effective modulator therapies, particularly the opportunities of initiating treatment early, by referencing the use of ivacaftor in people with gating mutations.
Identify future challenges in ensuring that highly effective treatments are available to all people with CF.
M.D., MBChB, MRCP,
Prof - Paed Respirology & Experimental Medicine
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