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"Entering the Era of Highly Effective Modulator Therapy"

November 1, 2019

In this session, Professor Jane Davies from the National Heart & Lung Institute, Imperial College in London, describes the progress and the promise of highly effective CFTR modulator therapy for people with CF. She highlights the long-term benefits such therapies have 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 uses 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 is illustrated with pre-school and infant data. 

Download Presentation Files
Jane C. Davies, M.D. PowerPoint PDF
Topics: Health Outcomes, CFTR, Plenary
Related: Plenary Session II: Friday, November 1, 2019