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"Matching Medicines With Mutations"

Thursday, November 2, 2017

Increasing numbers of people with CF now benefit from disease-altering small molecule therapies that target the basic defect in CFTR.  Use of these therapies has been extended recently using a combination of in vitro laboratory experiments to identify responsive mutations, and clinical trials to determine effect on clinical outcomes. This plenary will summarize how recent advances may allow in the near future over 90% of individuals with CF to benefit from therapies that target the basic defect in CFTR, including even those with the rarest disease causing mutations.

Download Presentation Files
Michael P. Boyle, M.D., FCCP PowerPoint PDF
Philip J. Thomas, Ph.D. PowerPoint PDF
Topics: Health Outcomes, Treatment, CFTR, Plenary, Clinical Research, Drug Development Pipeline
Related: Plenary Session II: Friday, November 3, 2017