Evaluating Effects of CFTR Modulation in Cystic Fibrosis

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Presentation transcript:

Evaluating Effects of CFTR Modulation in Cystic Fibrosis

Cystic Fibrosis A Genetic Disease

Six Classes of CFTR Gene Mutations

CFTR Mutation Classification

Six Classes of CFTR Gene Mutations

Modulators, Potentiators, Correctors

Ivacaftor (Potentiator) in G551D Gating Mutation

Ivacaftor (Potentiator) Plus Lumacaftor (Corrector) in F508del CFTR TRAFFIC and TRANSPORT

Considerations for CFTR Modulator Therapy in Children Younger Than 12 y

Ivacaftor in Children Aged 6 to 11 y

Ivacaftor in Very Young Children (2 to 5 y) KIWI

Ivacaftor in Non-G551D Gating Mutations KONNECTION

Medical Management of CF

Use of Ivacaftor in Children

Use of Lumacaftor-Ivacaftor in Adults Baylor Experience – Patient Selection

Lumacaftor-Ivacaftor in Adults Baylor Experience

Abbreviations

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