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31st Annual North American Cystic Fibrosis Conference


P1 - P1: Matching Medicines With Mutations


Nov 2, 2017 4:30pm ‐ Nov 2, 2017 6:00pm

Description

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.

Learning Objectives:

  1. Define that although CF is caused by a number of different mechanisms, mutations can be grouped by the nature of the defect and responsiveness to small molecule therapeutics.
  2. Recall how laboratory experiments can and are being used to determine which rare disease-causing mutations are responsive to therapies.
  3. Recall 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.

Plenary Leader(s):

Speaker(s):

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