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- Plenary Session II: Friday, November 1, 2019
"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.
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